Agency officials promise fast reviews of new treatments while vowing they will not be a “rubber stamp” for the industry. But patients with rare diseases view recent decisions as signs that the doors are closing on their options.

Ollie and Dr. Simon Jones made it on BBC Breakfast for a feature with Fergus Walsh about gene therapy for his MPSII Hunter Syndrome.

Three-year-old Oliver Chu has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy.