This page lists active clinical trials for MPS II. This page serves as informative only, Project Alive does not endorse any particular clinical trial. To find additional information, visit www.clinicaltrials.gov and search "mucopolysaccharidosis II" and look for trials that are recruiting. Once you click into a study, you will find the clinical trial site information near the bottom with contact information. Reach out to the listed contact to discuss the details of the trial.

Summary report resulting from an externally conducted Patient-Focused survey Hunter syndrome (MPS II) Cognitive and Behavioral results from the Toileting Ability Survey

Julie Eisengart, PhD, LP, the Associate Professor of Pediatrics of the Neurodevelopmental Program in Rare Disease at the University of Minnesota, presents on Accommodating Behaviors in the Classroom & Clinic at the 2024 Hunter Syndrome Community Conference hosted by Project Alive.

This study examines the outcomes of enzyme replacement therapy (ERT) in siblings who began treatment at different ages. Findings suggest that earlier initiation of ERT may lead to milder disease progression and fewer somatic complications.

Lauren Newhouse, OTD, OTR/L, SWC, a pediatric occupational therapist, presents on Therapy for Neurodegenerative Disorders at the 2024 Hunter Syndrome Community Conference hosted by Project Alive.

Project Alive has funded the "Transitioning from Pediatric to Adult Health Care in Lysosomal Diseases: The MPS Experience" study. The study survey will be live soon. Please stay tuned for the study link. The attached file is the official study poster presented at WORLDSymposium in February, 2024.

NeuroJourney uses four phases of a full life to help you understand the challenges you might face, anticipate the conversations you may have, and prepare for the decisions you may need to make as you determine goals of care for your child.

This research examines how MPS II affects patients' health, well-being, and life expectancy, highlighting the significant burden the disease places on individuals and their families.

Phone: 800-737-3028 Email: CYSHCN.Helpline@dhhs.nc.gov The North Carolina Children and Youth with Special Health Care Needs Help Line is a free information and referral source for caregivers of and professionals who work with children and youth who have or are at risk for chronic physical, developmental, behavioral or emotional conditions, needing health services beyond the care that a child typically needs. We offer support on a range of topics such as: • Services available under government and community programs. • Resources to help pay for medications, equipment/supplies, adaptive devices, utilities or food. • Available health care and dental providers. • Information on how to find services when relocating to and within North Carolina. • Health care transitions, guardianship and alternatives to guardianship.

Phone: 919-707-5600 Genetic Counseling Services identify individuals and families who may have or be at risk of having a genetic condition. Services are focused on providing early intervention, education, diagnosis, and treatment if possible. Genetic counselors from the Children and Youth Branch routinely provide consultation to health care providers on many genetic issues. Genetic services are available at five medical centers and one community hospital in NC for any infant, child, or adult suspected of having a genetic condition. This also includes services for pregnant women that are at an increased risk to have a baby with birth defects. The state genetic counselor can assist with: • referral to genetic evaluations • genetic counseling • genetic educational materials • assistance locating support groups (for specific genetic conditions) Any individual with a potential genetic disorder is eligible for services. Families who meet financial eligibility are not billed for services.

Mercy Medical Angels aids in the air with commercial airline tickets and volunteer pilots, and on the ground with gas cards, bus and train tickets at no cost to patients. Mercy Medical Angel’s program Angel Flight Mid-Atlantic serves the residents of Virginia, Maryland, West Virginia, Pennsylvania, Tennessee, Kentucky, North Carolina, Delaware, Michigan, Ohio, and Washington, DC. Commercial Airline Tickets o Mercy Medical Angels provides charitable, need-based commercial airline assistance for patients and one medically necessary escort. We require:  A minimum of 21 days’ notice before your travel date to consider flight assistance.  A completed medical clearance form from the doctor who has recently physically seen the patient and knows they are going to this appointment. Our medical clearance form will be faxed to the doctor after we receive your application.  Income verification which includes total household income and assets for everyone living in the household. Forms of verification include current year complete income tax return and current award letters from Disability and SSI (if applicable).  Mercy Medical Angels may sponsor a round-trip for a patient and one non-medical attendant (if the doctor finds it medically necessary) on a commercial flight, coach seating. The patient must:  Be able to sit on their own once they are in the seat.  Require little or no medical attention.  Be able to transfer out of their wheelchair into their seat on the aircraft with little or no assistance (they cannot have someone literally pick them up and sit them in the seat).  If the patient requires oxygen, some airlines may provide it during the flight. Otherwise, the patient must have a portable oxygen concentrator (POC) approved by the FAA or the airlines. Commercial airlines require no less than 48 hours advance notice to prepare the equipment needed. The patient is responsible for making these arrangements and paying any travel fees associated with the oxygen.  An income-based, minimal, nonrefundable airline ticket processing fee will be determined upon verification of all required income documentation. Ground Transportation Mercy Medical Angels’ ground transportation program assists financially qualifying patients. o Patients are provided with gas cards, or commercial bus or Amtrak tickets to medical-related treatment. These trips are for non-local medical care more than 50 miles away. Gas cards are also available for multiple trips to local treatment. Patients (with or without escort) must be ambulatory and require no medical monitoring or care during the flight. Mercy Medical Angel’s program Angel Flight Mid-Atlantic serves the residents of Virginia, Maryland, West Virginia, Pennsylvania, Tennessee, Kentucky, North Carolina, Delaware, Michigan, Ohio, and Washington, DC.

Phone: 866-219-5262 Legal Aid of North Carolina is a statewide, nonprofit law firm that provides free legal services in civil matters to low-income people to ensure equal access to justice and to remove legal barriers to economic opportunity. With a holistic approach, Legal Aid of North Carolina provides free legal help when people need it most because everyone deserves an advocate when their home, health or livelihood is on the line.

Phone: 919-856-2195 Disability Rights North Carolina is a legal advocacy agency that fights for the rights of people with disabilities in North Carolina. We handle cases involving discrimination, abuse and other rights violations. All our services are at no cost to North Carolinians with disabilities. We are a private, independent, 501(c)(3) nonprofit and a member of the National Disability Rights Network.

A statewide information and referral service that can connect families with local, state, and federal resources for children with disabilities. You can call 211 or visit their website.

Phone:(877) 384-2575 Email: ACPSupport@usac.org The Affordable Connectivity Program (ACP) is a U.S. government program that helps many low-income households pay for broadband service and internet connected devices. You are likely eligible if your household’s income is below 200% of the Federal Poverty Line, or if you or someone you live with currently receives a government benefit like SNAP, Medicaid, SSI, WIC, Pell Grant, or Free and Reduced-Price Lunch. - If your household is eligible, you could receive: • Up to a $30/month discount on your internet service • Up to a $75/month discount if your household is on qualifying Tribal lands • A one-time discount of up to $100 for a laptop, tablet, or desktop computer (with a co-payment of more than $10 but less than $50) • A low-cost service plan that may be fully covered through the ACP Only one monthly service discount and one device discount is allowed per household. To receive the connected device discount, consumers need to enroll in the ACP with a participating provider that offers connected devices (Note: not all internet company offer device discounts.) The internet company will provide the discount to the consumer.

Phone: (202) 454-3970 Email: info@caregiveraction.org Caregiver Action Network (CAN) is the nation’s leading family caregiver organization working to improve the quality of life for the more than 90 million Americans who care for loved ones with chronic conditions, disabilities, disease, or the frailties of old age. CAN serves a broad spectrum of family caregivers ranging from the parents of children with significant health needs, to the families and friends of wounded soldiers; from a young couple dealing with a diagnosis of MS, to adult children caring for parents with Alzheimer’s disease. CAN (the National Family Caregivers Association EIN 52-1780405) is a non-profit organization providing education, peer support, and resources to family caregivers across the country free of charge.

Rare Caregivers is a comprehensive online guide that addresses the distinct needs of family caregivers of loved ones with rare diseases. Rare Caregiver’s resources provide caregivers with tips on helping their loved ones as well as themselves.

Phone: 800-852-0042 Email: fsp.cdr@unc.edu “Family Support” helps families with a child who has special needs access services and supports. Family support is a community-based approach that… o Increases the strength and resilience of families o Supports parents as they help their children with special needs achieve their full potential o Improves community providers’ capacity to serve families with children who have special needs All FSN Regional Affiliates Offer: o Parent-to-Parent Support o One-to-one emotional and informational support for parents with a child who has special needs by a trained Parent Mentor. Pairing parents of newly diagnosed children with experienced, trained support parents helps families adjust, adapt, and feel supported. - Information and Referral o Regional FSN affiliates provide families and service providers with information and referrals to community and regional services and resources to help children and their families. Support Groups and Workshops o Provides social and emotional support, information and skills training, and leadership opportunities for families. Parent to Parent Matching for Rare Disorders

These centers provide free training to families about special education laws and services available to children with disabilities. In North Carolina, the Exceptional Children's Assistance Center (ECAC) is the state's PTI, offering resources, workshops, and one-on-one assistance.

Phone: 800-318-8861 Healthcare Hospitality Network, Inc. (HHN) is a nationwide professional member association with a mission to support homes that help and heal to be more effective in their service to patients and families. The HHN community includes House members and Supporting Partner members. Our House members provide lodging and support services to patients and their caregivers while they are receiving medical treatment away from their home communities. Our Supporting Partners are members that support the Healthcare Hospitality Houses in their services to their guests. HHN is the only professional membership association in North America with the specific mission to provide training, networking, and advocacy specifically for healthcare hospitality houses. Founded in the early 1980s and incorporated in 1986, HHN proudly serves over 100 member houses ranging from small houses to houses with over 100 guest rooms. Supporting Partners include Trivers Associates, Non-Profit Software, and Palmetto Linen.

Lasagna Love is a global nonprofit and grassroots movement that aims to positively impact communities by connecting neighbors with neighbors through homemade meal delivery. We also seek to eliminate stigmas associated with asking for help when it is needed most. Our mission is simple: feed families, spread kindness, and strengthen communities.

American Bar Association Free Legal Answers is a virtual legal advice clinic in which qualifying users can post a civil legal question to be answered by pro bono attorneys licensed in their state. Question topics include family, divorce, custody, housing, eviction, homelessness, consumer rights, finance, work, employment, unemployment, health and disability, civil Rights, income maintenance, juvenile and education law. By using this website, you are asking a lawyer to give you legal help on your specific legal questions. They will be your lawyer for this limited purpose and will not do anything for you other than provide this help. The lawyer will answer your questions through the website and does not have to separately email you an answer or have a phone call or meeting to answer your questions. If the lawyer leaves your question open for a response from you, you will have 10 days to reply before the question closes. When the lawyer is finished answering your question, the lawyer will no longer be your lawyer. If you have a court date or you need to file something with the court by a specific date, a lawyer may not answer your question in time. You should keep looking for a lawyer while you wait to see if a lawyer will answer your question. Unfortunately, not all questions posted to the site will receive an answer. If an attorney cannot assist you, an administrator will notify you. You can use the website to ask 3 different legal questions per year. You can’t create a new account to ask additional questions. You must meet our rules every time you use the website. Eligibility You can use this website IF all of these are true for you: • Your income is low for a family the size of yours. • The value of your checking account, savings account, stocks or bonds are low. • You aren’t in jail or prison. • Your legal problem isn’t related to a criminal charge. • You don’t already have a lawyer to help you with your legal problem. • You are an adult.

Global Genes’ story began with our loved ones. We are the friends, family and supporters of patients close to us who are affected by rare disease. We understand the confusion, the overwhelming experience of the unknown and the feelings of isolation all too well. And we know we aren’t alone. That’s the reason Global Genes was born – to connect, empower and inspire the rare disease community. Our mission is to provide patient advocates with a continuum of services to accelerate their path from early support and awareness through research readiness, using a collaborative approach that involves biopharma, researchers and funders, with data as a central core.

TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists are administered on a calendar-year basis. At the end of each calendar year, waitlist applications expire. Patients still seeking assistance must join waitlists for the subsequent calendar year.

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures. We do not speak for patients. We provide the training, education, resources and opportunities to make their voices heard. By activating the patient advocate, we can change public policy and save lives.

Family Voices is a national family-led organization of families and friends of children and youth with special health care needs (CYSHCN) and disabilities. We connect a network of family organizations across the United States that provide support to families of CYSHCN. We promote partnership with families at all levels of health care–individual and policy decision-making levels—to improve health care services and policies for children.

310-451-1353 866-451-5367 admissions@thepaintedturtle.org The Painted Turtle is more than a camp. It’s a place where children with serious medical conditions can just be kids. Totally free of charge. The mission of The Painted Turtle is to provide a year-round, life-changing environment and authentic camp experience for children with chronic and life-threatening illnesses. The Painted Turtle supports children’s medical needs, inspires them to reach beyond their illnesses, and provides care, education, and respite for their families. Requirements and Eligibility • Age requirements: o Family Weekend: Between the ages of 6-17 o Summer Camp: Between the ages of 7-16 o Have a minimum developmental age of 5 years • Be able to function and participate in a group setting • Able to communicate needs independently • Can be without family members for a limited period of time (overnight retreat and family weekends) or the duration of the camp session (summer camp). Please be advised that no child is guaranteed to be accepted to camp. Applications are reviewed and acceptance is based upon health care needs, space, and staffing ratios.

LawHelpCA has been prepared for general information purposes only. It does not provide direct legal advice but can connect you to legal aid organizations and referral services. What is LawHelpCA? • LawHelpCA is California’s official and free legal resource. • Reliable information about common legal issues, written by reputable sources on 16 general topics and over 140 specific subtopics • Updated directory of legitimate and pre-screened organizations that offer free or low-cost legal advice and representation

Need help paying your hospital bill? You may be eligible for financial assistance. Most hospitals offer discounts and sometimes free case to those who cannot afford to pay. Your eligibility usually depends on your household income and the number of people who live in your household. This is compared to the federal poverty level and is referred to as the “percent of federal poverty level” (% FPL). To learn more about your hospital’s discount policy: • Find your hospital in the list (shown alphabetically, by county). • In the column POLICY, click on the icon to view, download, or print the hospital policy. • In the column APPLICATION, click on the icon to view, download, or print the application form you may need to complete. • Contact the hospital to find out what you need to qualify (contact information is usually in the DETAILS).

(916) 445-8949 access@parks.ca.gov The California Department of Parks and Recreation, popularly known as State Parks, is honored to provide access to parks and open spaces and contribute to a healthier and richer quality of life for Californians and for people all over the world who visit the golden state’s natural wonders. For persons with permanent disabilities, the Disabled Discount Pass entitles the bearer to a 50% discount for vehicle day use, family camping, and boat use fees at over 100 California State Park operated units. There is a $3.50 application fee to apply for the Disabled Discount Pass. How to obtain the Disabled Discount Pass 1. Apply and submit your documents, including payment. 2. Get approved. 3. Receive your pass. 4. Enjoy discounted entry to the parks.

(323) 222-8090 The Diagnostic Center, Southern California provides high-quality, individualized services to special education students, their families, and school districts. Services are provided by expert, transdisciplinary teams of diagnostic professionals that may include educational specialists, speech-language specialists, school psychologists, clinical psychologists, motor specialists, and pediatricians who address the unique educational needs of Southern California’s most difficult-to-serve students enrolled in special education programs. The Diagnostic Center, Southern California is one of three regional assessment centers operated by the State Special Schools and Services Division of the California Department of Education. The Centers provide assessment, training, and technical assistance to all Local Educational Agencies (LEAs) in California. Each of the three Centers serves a specific geographic region in the state: Southern California-Los Angeles, Northern California- Fremont, Central California-Fresno. Our services include: • Comprehensive, state-of-the-art assessment and educational planning services to assist local school districts in determining the needs of their most complex students. • On-site technical assistance and consultation in program and instructional design based on the unique needs of each student. • Professional development opportunities for teachers, administrators, and special education staff, including presentations at national, statewide, and local conferences and workshops, provided by specialists with “hands-on” experience. • Family education opportunities, including consultation, referral, and resource information. There is no cost for assessment services; however, requests for services must be generated by the local school district.

(916) 441-5844 info@cal-dsa.org The mission of the California Disability Services Association (CDSA) is to advocate for and strengthen member organizations to ensure public and government support of services for people with intellectual or developmental disability (IDD). CDSA is a statewide association representing more than 110 community-based organizations that support and empower people with intellectual and developmental disabilities and their families. Our members create choices and opportunities that help clients lead the lives they choose and reach the goals they set. Their programs help clients develop skills, engage in meaningful activities, participate in community life, and live independently. Their services also help family caregivers balance life’s demands.

Founded in 1984, the California Caregiver Resource Centers are a network of 11 centers throughout California which serve family caregivers who are providing support for someone affected by chronic and debilitating health conditions including dementia, Alzheimer’s disease, cerebrovascular diseases (such as stroke or aneurysms), degenerative diseases such as Parkinson’s, Huntington’s and multiple sclerosis, or traumatic brain injury (TBI), among many others.

California Action Link for Rare Diseases (CAL RARE) is dedicated to improving the lives of rare disease patients in California. We are a coalition of rare disease stakeholders with a goal to raise awareness among the public and decision makers regarding rare diseases. We want to bring recognition to the issues rare disease patients face and work to ensure access to physicians, treatments, and social services.

424-334-1773 info@awalkonwater.org Since launching in 2012, A Walk on Water (AWOW) has served thousands of children with unique needs, and their families through transformative Surf Therapy. AWOW Surf Therapy is uniquely built on the concept of including the entire family in a connection to the healing powers of the ocean and our natural world. By creating positive, uplifting, and empowering experiences, we are rewriting the expectation of what “therapy” can be. Surf Therapy is generally defined as the act of guided surf instruction provided by highly trained Surf Instructors, in concert with a carefully constructed and supportive team of volunteers and staff. While Surf Therapy is the core offering of AWOW and the driving force of our efforts, our events offer additional elements that add to the ability to feel fulfilled and rejuvenated. Some of the offerings include yoga, massage therapy, art therapy, music therapy, food and drink, and many different variations of sport and play on the beach. We consider ourselves–our volunteers, partners, and participants–one big family: Our AWOW Family. By maintaining a high ratio of volunteers to athletes, AWOW has built a platform that allows each participant the chance to bond with fellow athletes and volunteers, forming meaningful, lasting relationships. These lifelong bonds are vital to developing the confidence necessary for each child to succeed not only in the highly difficult skill of surfing but more importantly, in navigating the waters of life. Diligently, we work to enable our amazing athletes to realize confidence in themselves. We’re equipping our participants to defy expectations, rewrite their futures, and emerge from the ocean with one belief; we are all ATHLETES.

(510) 533-1248 Provides comprehensive healthcare services, including support for housing stability.

Call 510-238-6182 for assistance. Services: Offers guidance on affordable housing options and tenant rights.

Call 510-238-3721 or email RAP@oaklandca.gov. Provides information on tenant rights and rent stabilization ordinances.

Offers car-sharing services with multiple locations in Oakland.

310-390-2958 info@angelflightwest.org Angel Flight West provides air transportation within and across only 13 Western states: Alaska, Arizona, California, Colorado, Hawaii, Idaho, Montana, Nevada, Oregon, Utah, Washington, and Wyoming. Although Angel Flight West responds to a wide variety of needs, flights are generally for passengers who can’t afford airline tickets, or for whom travel by land or by commercial airline would pose a physical hardship. Other viable flight situations include passengers with compromised immune systems or who live in remote areas without access to commercial transportation.

Lasagna Love is a global nonprofit and grassroots movement that aims to positively impact communities by connecting neighbors with neighbors through homemade meal delivery. We also seek to eliminate stigmas associated with asking for help when it is needed most. Our mission is simple: feed families, spread kindness, and strengthen communities.

American Bar Association Free Legal Answers is a virtual legal advice clinic in which qualifying users can post a civil legal question to be answered by pro bono attorneys licensed in their state. Question topics include family, divorce, custody, housing, eviction, homelessness, consumer rights, finance, work, employment, unemployment, health and disability, civil Rights, income maintenance, juvenile and education law. By using this website, you are asking a lawyer to give you legal help on your specific legal questions. They will be your lawyer for this limited purpose and will not do anything for you other than provide this help. The lawyer will answer your questions through the website and does not have to separately email you an answer or have a phone call or meeting to answer your questions. If the lawyer leaves your question open for a response from you, you will have 10 days to reply before the question closes. When the lawyer is finished answering your question, the lawyer will no longer be your lawyer. If you have a court date or you need to file something with the court by a specific date, a lawyer may not answer your question in time. You should keep looking for a lawyer while you wait to see if a lawyer will answer your question. Unfortunately, not all questions posted to the site will receive an answer. If an attorney cannot assist you, an administrator will notify you. You can use the website to ask 3 different legal questions per year. You can’t create a new account to ask additional questions. You must meet our rules every time you use the website. Eligibility You can use this website IF all of these are true for you: • Your income is low for a family the size of yours. • The value of your checking account, savings account, stocks or bonds are low. • You aren’t in jail or prison. • Your legal problem isn’t related to a criminal charge. • You don’t already have a lawyer to help you with your legal problem. • You are an adult.

Advocates for resources, funding, and services to help individuals with developmental disabilities live fulfilling lives in their communities.

American Bar Association Free Legal Answers is a virtual legal advice clinic in which qualifying users can post a civil legal question to be answered by pro bono attorneys licensed in their state. Question topics include family, divorce, custody, housing, eviction, homelessness, consumer rights, finance, work, employment, unemployment, health and disability, civil Rights, income maintenance, juvenile and education law. Eligibility You can use this website IF all of these are true for you: • Your income is low for a family the size of yours. • The value of your checking account, savings account, stocks or bonds are low. • You aren’t in jail or prison. • Your legal problem isn’t related to a criminal charge. • You don’t already have a lawyer to help you with your legal problem. • You are an adult.

Angel Flight is a non-profit organization consisting of a network of volunteer pilots who donate their time and aircraft to provide free private non-emergency air transportation for the medically and financially needy to distant medical institutions. Note Angel Flight is not an air ambulance. Patients must be ambulatory, meaning they must be able to walk independently, enter and exit the plane with little or no assistance and require no medical care en route. There are several branches of Angel Flight. The state of Wisconsin is served by Angel Flight Central along with Kansas, Missouri, Iowa, Nebraska, Illinois, Minnesota, Indiana, South Dakota and North Dakota.

312-939-3513 info@frcd.org The Family Resource Center on Disabilities (FRCD) stands as a beacon of support and empowerment for families in the Chicago Metropolitan Area who have children with disabilities. Dedicated to ensuring that every child receives the quality education and services they deserve, FRCD offers a wealth of resources and guidance to parents navigating the intricate landscape of special education. Serving eight Illinois counties – Cook, DuPage, Grundy, Kane, Kendall, Lake, McHenry, and Will – FRCD is a vital partner in the journey towards providing children with disabilities the opportunity to lead productive and independent adult lives.

The Division of Developmental Disability Services sponsors programs designed to provide individuals with developmental issues with services designed to address their needs. Partner community agencies provide services and support for individuals with developmental disabilities to help these individuals achieve as much independence as possible.

Provides comprehensive disability services, including therapy, education, family support, and respite care for families caring for children with special needs.

Lasagna Love is a global nonprofit and grassroots movement that aims to positively impact communities by connecting neighbors with neighbors through homemade meal delivery. We also seek to eliminate stigmas associated with asking for help when it is needed most. Our mission is simple: feed families, spread kindness, and strengthen communities.

Phone: (202) 454-3970 Email: info@caregiveraction.org Caregiver Action Network (CAN) is the nation’s leading family caregiver organization working to improve the quality of life for the more than 90 million Americans who care for loved ones with chronic conditions, disabilities, disease, or the frailties of old age. CAN serves a broad spectrum of family caregivers ranging from the parents of children with significant health needs, to the families and friends of wounded soldiers; from a young couple dealing with a diagnosis of MS, to adult children caring for parents with Alzheimer’s disease. CAN (the National Family Caregivers Association EIN 52-1780405) is a non-profit organization providing education, peer support, and resources to family caregivers across the country free of charge.

Rare Caregivers is a comprehensive online guide that addresses the distinct needs of family caregivers of loved ones with rare diseases. Rare Caregiver’s resources provide caregivers with tips on helping their loved ones as well as themselves.

TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists are administered on a calendar-year basis. At the end of each calendar year, waitlist applications expire. Patients still seeking assistance must join waitlists for the subsequent calendar year.

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures. We do not speak for patients. We provide the training, education, resources and opportunities to make their voices heard. By activating the patient advocate, we can change public policy and save lives.