-
Educating all ages about living with muscular dystrophy
In my four decades of life with limb-girdle muscular dystrophy, I’ve become comfortable educating everyone I meet about my quality of life and the ways all of us living with chronic, rare conditions have been given a wonderful chance to influence societal views of the disabled community. I’ve found it most effective to keep my…
-
C-Path to lead new task force seeking to advance LGMD therapies
The Critical Path Institute (C-Path) is launching a task force to advance the development of therapies for limb-girdle muscular dystrophy (LGMD) and will lead the efforts of its members to find a new treatment to, among other goals, slow or stop the progression of the muscle-wasting disease. Formed under the nonprofit’s Rare Disease Cures Accelerator-Data…
-
This time, new medical equipment is hard for me to accept
Our house is getting a new piece of medical equipment this week. That’s happened before, as we have three sons with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. Over the past decade, in fact, our house has been a landing zone for lots of equipment as we’ve adapted to their disease…
-
Rare pediatric disease designation granted to LAMA2-CMD therapy
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to MDL-101, an epigenetic editing therapy that Modalis Therapeutics is developing for LAMA2-related congenital muscular dystrophy (LAMA2-CMD) — a genetic condition that’s characterized by muscle wasting evident at birth or shortly thereafter. This FDA status recognizes MDL-101 for its potential to offer…
-
WATCH: Interim Results from FORWARD-53 Study of WVE-N531 in Duchenne Muscular Dystrophy (Webinar Recording)
Wave Life Sciences recently joined PPMD for a presentation of interim results from the company’s ongoing Phase 2 FORWARD-53 study of WVE-N531 in individuals living with Duchenne muscular dystrophy who are amenable to exon 53 skipping, first released on September 24, 2024. Wave’s President and CEO and Chief Development Officer highlighted that WVE-N531 demonstrated substantial…
-
DMD treatment WVE-N531 boosts dystrophin: Interim trial data
Treatment with exon 53-skipping therapy WVE-N531 led to significant increases in muscle dystrophin levels along with signs of improved muscle health and regeneration in boys with Duchenne muscular dystrophy (DMD). That’s according to new six-month interim data from the Phase 1b/2a FORWARD-53 trial (NCT04906460), which also found the treatment to be safe and well tolerated.…
-
2024 Rev It Up 5k Raises $27,000 for PPMD
The Rev It Up 5K presented by Mitsubishi Electric Automotive America held their 4th annual event on September 21, 2024, bringing in $27,000 for PPMD! Thank you to Jason Dempsey for organizing this event and making it incredibly fun and memorable for so many. It would not have been the success that it was without…
-
PPMD Hosts Meeting Exploring the Case for Cardiac Transplantation in Duchenne
As part of our ongoing Cardiac Initiative, PPMD is proud to host the upcoming professional meeting, “Expanding Access to Heart Transplantation: Focus on Muscular Dystrophy,” taking place September 27-28, 2024, at Stanford University. This important event will bring together leading experts in cardiology, neuromuscular disorders, and transplant medicine to address a critical but often overlooked…
-
Capricor to seek deramiocel’s FDA approval for DMD heart disease
Capricor Therapeutics will soon begin filing an application seeking U.S. approval of its cell therapy deramiocel for cardiomyopathy, a disease of the heart’s muscle, in people with Duchenne muscular dystrophy (DMD). Following recent meetings with the U.S. Food and Drug Administration (FDA), Capricor will file a rolling application, meaning the company can submit parts of…
-
PPMD Joins Duchenne Parent Project aps Respiratory International Professional Care Meeting
Parent Project Muscular Dystrophy (PPMD) is pleased to announce our participation in the upcoming “Respiratory Assessment and Management of Duchenne Muscular Dystrophy: Current Standards and Future Directions” meeting, which will take place in Rome, Italy, from September 26–27, 2024. This important event will bring together experts from around the world to discuss advancements in respiratory…
