Uncategorized

Over the past three decades, PPMD has driven Congress to enact legislation that has transformed federal public health, research, therapy development and access policies to bring us closer to ending Duchenne. This advocacy work has resulted in marked increases in Duchenne and related muscular dystrophy research funding, thorough public health surveillance to better understand the size…

Know More

The PPMD Town Hall on Clinical Trials, taking place on Monday, March 4th during PPMD’s 2024 Advocacy Conference in Washington, D.C., aims to provide a platform for families affected by Duchenne and Becker to share their experiences and insights, with a focus on past, present, and future clinical trials. The primary goal is to hear…

Know More

I’m reading “Eyes to the Wind: A Memoir of Love and Death, Hope and Resistance” by Ady Barkan, a late lawyer and political activist who had ALS. It’s been a tough read so far. In the book, Barkan recounts his experiences after being diagnosed with ALS in his early 30s. He does an excellent job…

Know More

Life is lived in seasons, at least here in Nebraska, where we experience all four of them. Some days offer nearly perfect weather: The sun is shining, the temperature is in the mid-70s, and I can smell the thaw of winter and feel the hope of spring. Then there are the cloudy winter days when…

Know More

Patients and experts in limb-girdle muscular dystrophy (LGMD), along with drug developers, community leaders, and regulatory agency personnel, came together for the LGMD Scientific Workshop to discuss how to tackle the unmeet therapeutic needs of people with these muscle-wasting diseases. The workshop, held in early February in Maryland, was hosted by The Speak Foundation, a…

Know More

 “Disco For Duchenne” Gala Features Dancing, Drinks, a Culinary Experience and More  to Help Advance Research for the Rare Disease  AUSTIN, Texas., February 28, 2024 – CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy, and the Revell family of Austin will host the 15th annual fundraiser gala, Champions…

Know More

Dyne Therapeutics will present initial clinical data, first released on January 3rd, 2024, from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Recorded Tuesday, February 13, 2024 Watch Recording The post CureDuchenne Webinar with Dyne Therapeutics | Advancing the Promise of FORCE to Deliver…

Know More

A cell-based study revealed the biological mechanism behind the increased tendency for people with myotonic dystrophy type 2 (DM2) to develop autoimmune diseases. Researchers found that the genetic defect that causes DM2, called a repeat expansion, indirectly triggered an abnormal, antiviral immune response in patient cells. “That was our ‘aha’ moment,” study lead Claudia Günther,…

Know More

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…

Know More

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…

Know More