WebOct 1, 2024 · NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the U.S. Food and Drug Administration (FDA). PF-06939926 is currently being evaluated to … WebApr 11, 2024 · RGX-202 is currently being evaluated in the phase 1/2 AFFINITY DUCHENNE clinical trial (NCT05693142), which is actively recruiting patients with DMD. …
PFIZER RECEIVES FDA FAST TRACK DESIGNATION FOR DUCHENNE MUSCULAR ...
WebJul 27, 2024 · Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy. WebDuchenne muscular dystrophy has X-linked recessive inheritance, but about 30% of cases happen spontaneously without a family history of the condition. X-linked means the gene responsible for DMD is located on the X chromosome, one of two sex chromosomes. expectations of employees policy
FDA Staff Had Inclination Towards Rejecting Sarepta …
WebJan 11, 2024 · Request an appointment phone 410-955-4259 Nance explains that DMD is caused by deletions, duplications, point mutations or premature stop codons in the gene … Web23 hours ago · FDA designation comes as AFFINITY DUCHENNE study is underway. by Marisa Wexler, MS April 13, 2024. The U.S. Food and Drug Administration (FDA) has granted fast track designation to RGX-202, a one-time gene therapy for Duchenne muscular dystrophy (DMD) that is in early clinical trials. The FDA gives this designation … WebMar 21, 2024 · There is no current cure for Duchenne muscular dystrophy (DMD), a rare genetic disease in young male patients, and the aim of treatment is to delay disease progression. Recent advancements in gene therapy provide potential improvement in targeting the underlying cause of DMD. Since 2016, the US FDA has approved four new … expectations of employees examples