Duchenne Muscular Dystrophy Cure
Gene Therapy: Behind the Scenes
Unlocking the Promise of Gene Therapy
Pfizer is working to unleash the next wave of treatment innovation for patients living with rare genetic diseases.
Sarepta Therapeutics Announces FDA Approval of ELEVIDYS
ELEVIDYS addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as ELEVIDYS micro-dystrophin.
Duchenne Muscular Dystrophy
An inherited disorder of progressive muscular weakness, typically in boys.
Many people with muscular dystrophy have Duchenne syndrome. Girls can be carriers and mildly affected, but the disease typically affects boys.
Rare Disease Day
Raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families and carers.
Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact.