The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an investigational gene therapy for limb-girdle muscular dystrophy type 2E (LGMD2E). “This is one of the first programs to receive platform technology designation and an important recognition by FDA of…
I believe that many adults living with rare diseases can and do become experts in their conditions, sometimes more so than healthcare professionals. With respect to my own journey with limb-girdle muscular dystrophy, this point became particularly clear when I had a near-death experience in early 2024. Several types…
When I think about my son Charlie, I always remember my pregnancy with him. I’d taken a home test and knew I was pregnant, but when I called to make an appointment with my doctor to confirm the good news, I scheduled an appointment for my son Max, who was…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart’s experimental stem cell therapy for heart problems in Duchenne muscular dystrophy (DMD), the company announced in an email sent to Muscular Dystrophy News Today. This FDA designation aims to accelerate the development…
Last Thursday, I participated in a workshop that used experiential theater to examine healthcare conversations. With a sense of anticipation and hope, I stepped into the unknown, eager to gain insights that could enrich my advocacy for Duchenne muscular dystrophy (DMD), which I have. The workshop, held by the…
Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental exon 51-skipping therapy for Duchenne muscular dystrophy (DMD). The company said it will wind down all DMD-related research and development activities, focusing instead on an investigational therapy for myotonic dystrophy type…
During a recent facioscapulohumeral muscular dystrophy (FSHD) wellness group meeting via Zoom, we each shared how we were feeling about our disease journey. One participant said, “I feel like I’m always in transition.” I can relate to that. With FSHD, the need to adapt seems never-ending, a topic that…
Duchenne muscular dystrophy (DMD) is a rare genetic disorder that causes progressive muscle weakness. People living with DMD and their families face a variety of challenges every day. A path to prompt, affordable treatment should not be one of them. That is why Catalyst Pharmaceuticals created Catalyst Pathways®,…
Once again, I’m writing from an uncomfortable chair in an infusion room as my son Max participates in a clinical trial. I frequently visit this room with my three sons — Max, 19; Rowen, 16; and Charlie, 14 — who live with Duchenne muscular dystrophy (DMD). It’s a…
CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new gene therapy for Duchenne muscular dystrophy (DMD) that aims to overcome the limitations of existing gene therapies. “This investment underscores our continued use of venture philanthropy to catalyze progress [toward] transformative treatments…
Get regular updates to your inbox.