-
Breaking news: Rare Disease Day Congressional Hearing to Address the BENEFIT Act
On Thursday, February 29th, Rare Disease Day, the House Energy and Commerce Committee will hold a congressional hearing, titled “Legislative Proposals to Support Patients with Rare Diseases” at 10:00 am ET. This hearing will review 18 pieces of legislation intended to support patients living with rare diseases, including the Better Empowerment Now to Enhance and…
-
In choosing a college, my son with DMD is already winning
I am a competitive former college athlete married to a someone who is equally competitive and also was a college athlete. We like to win and try to position ourselves in ways to make it possible. However, when three of our sons, Max, 18, Rowen, 15, and Charlie, 13, were diagnosed with Duchenne muscular dystrophy…
-
Revised Community-Led Duchenne Guidance Published: Developing Potential Treatments for the Entire Spectrum of Disease
Today PPMD is pleased to share that the revised community-led Duchenne Guidance has been published in the Journal of Neuromuscular Diseases! This Community Draft Guidance was submitted to the FDA at the end of 2022. Serving as an update to the original guidance that was submitted in July 2014, the revision of this document is…
-
Exon-skipping AOC 1044 gets FDA rare pediatric disease status
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AOC 1044, an experimental therapy for people with Duchenne muscular dystrophy (DMD) caused by mutations that are amenable to exon 44 skipping. The FDA gives the designation to treatments with the potential to improve care for disorders that primarily affect children…
-
Watch: Overview of WVE-N531 and the FORWARD-53 Clinical Trial with Wave Life Sciences (Webinar Recording)
Wave Life Sciences recently joined PPMD for a community webinar to share an update on the company’s FORWARD-53 study. Wave provided an overview of initial clinical trial results for WVE-N531 in individuals with Duchenne muscular dystrophy who are amenable to exon 53 skipping, as well as an overview of the potentially registrational FORWARD-53 study. The…
-
I’m tapering off antidepressants and steroids with no regrets
On Feb. 19, I went to see my psychiatrist for the first time in 2024, after enduring a rocky start to the new year. When I last saw him in November, we’d decided that I’d consult him again as needed, but I wouldn’t have fixed appointments. The year started for me where 2023 had left…
-
SAT-3247 improves muscle function in FSHD mouse model
SAT-3247, an oral therapy candidate for rebuilding muscle tissue, improved muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD). Developed by Satellos Bioscience, the therapy is the lead treatment candidate for Duchenne muscular dystrophy (DMD), with the first clinical trial of SAT-3247 for DMD expected to start this year. “We look forward to…
-
PPMD Announces Pediatric Certified Duchenne Care Center at Penn State Health Children’s Hospital
Today PPMD announced the expansion of our renowned Certified Duchenne Care Center (CDCC) Program with the certification of the clinic at Penn State Health Children’s Hospital in Hershey, Pennsylvania. This designation marks a significant step for the CDCC Program and continues the growth and expansion to bring comprehensive care to all people living with Duchenne…
-
Sarepta Therapeutics Announces U.S. FDA Acceptance of an Efficacy Supplement to Expand the ELEVIDYS Indication
The FDA will make a decision whether or not to grant full approval of Sarepta’s gene therapy, Elevidys, by June 21,2024. The Efficacy Supplement submitted by Sarepta seeks to broaden the indication for Elevidys to all ages and irrespective of ambulation status. Read the press release: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-us-fda-acceptance-efficacy The post Sarepta Therapeutics Announces U.S. FDA Acceptance…
-
The things we don’t talk about as Duchenne caregivers
When three of my sons were diagnosed with Duchenne muscular dystrophy more than 10 years ago, I shared everything we experienced on social media and found support and understanding. But as they grow older, weaker, and more dependent on me, I find caregiving harder than I could’ve imagined. I want others to understand our life…
