March 2024

Italfarmaco S.p.A. has announced that the full results from the pivotal phase 3 EPIDYS clinical trial of givinostat in ambulant Duchenne patients six years of age and older have been published in The Lancet Neurology. Givinostat is Italfarmaco’s proprietary histone deacetylase (HDAC) inhibitor for the potential treatment of Duchenne. The EPIDYS clinical trial met its…

Know More

I am traveling this week with my three sons who have Duchenne muscular dystrophy (DMD): Max, 18; Rowen, 15; and Charlie, 13. The four of us are in Denver for three days of clinical trial appointments and one day of cardiology appointments. Other Duchenne moms are often surprised that I can travel with my nonambulatory…

Know More

Open The Duchenne Registry app and you will notice a new survey on Gene Therapy. If you do not see the Gene Therapy survey listed under the Activities section, please update your app. We recently added this new survey to collect data on who is receiving gene therapy, which gene therapy they received, whether the…

Know More

NS Pharma recently joined PPMD for a community webinar to share an overview of two of the company’s new exon skipping trials: Brogidirsen (exon 44) and NS-050/NCNP-03 (exon 50). Vamshi Rao, MD, discussed these two new clinical trials for individuals with Duchenne who are amenable to either exon 44 or exon 50 skipping. The NS…

Know More

DYNE-251 and DYNE-101, Dyne Therapeutics’ investigational therapies for forms of muscular dystrophy, appear safe and are showing signs of preliminary efficacy in Phase 1/2 clinical trials. These proof-of-concept data, announced by Dyne in January, were discussed in two oral presentations at the recent 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. The DELIVER trial…

Know More

The investigational cell therapy CAP-1002 has continued to slow declines in upper limb and heart function among boys and young men with Duchenne muscular dystrophy (DMD), according to two-year results from the HOPE-2 open label extension (OLE) study. The findings were presented at the recent 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference by…

Know More

As I write this column, my wife, Wendy, is undergoing abdominal surgery. I won’t go into details, but it’s a pretty extensive procedure, so I’m concerned. I’d like to be there for Wendy, but due to my health issues, it’s just not possible. Instead, my daughter Jill is with her mom at the hospital. Wendy…

Know More

This pre-recorded webinar delves into the world of travel with special guests, Marissa Penrod and Adam Malone. Not only is Marissa an expert on traveling with Duchenne, but Adam is a travel advisor who also happens to live with the condition. We are also be joined by Alan Chaulet, who is the Vice President of…

Know More

Celebrate three decades of progress, community, and resilience at PPMD’s 30th Annual Conference, June 27–29, 2024 in Orlando, Florida! Back in 1994, our very first Conference was held in Orlando, and now, 30 years later, we are excited to return and commemorate the tremendous strides we’ve made together. We invite you to join us as…

Know More

PPMD is pleased to learn of the U.S. commercial launch of AGAMREE® (vamorolone) oral suspension 40 mg/mL for the treatment of Duchenne in patients aged two years and older. According to Catalyst Pharmaceuticals, Inc., following the U.S. Food and Drug Administration (FDA) approval in October 2023, AGAMREE® is now available by prescription and dispensed throughout…

Know More