Parent Project Muscular Dystrophy

NEW YORK – November 05, 2026 -- The Race to End Duchenne team at the 2026 TCS New York City Marathon raised $300,000 for Duchenne and Becker muscular dystrophy research, care and advocacy. It is the most successful fundraising result in the history of the program's NYC Marathon effort, Parent Project Muscular Dystrophy (PPMD) announced.

Two runners finished the marathon in under three hours

PPMD's marathon team included two runners who ran sub-3:00 times: Nicholas Byrnes and Jack Napoli. Napoli is a longtime member of the Race to End Duchenne team. He finished in the top 1% of his age group (20-24) and in the top 1% of all marathon finishers.

United States – October 27, 2026 -- Families affected by Duchenne muscular dystrophy have early data on SGT-003, an investigational gene therapy from Solid Biosciences: 90 days after dosing, three boys showed average microdystrophin expression of 110%.

Three boys show muscle health improvements at 90 days

The Phase 1/2 INSPIRE DUCHENNE study reported initial data from its first three patients: two 5-year-old boys and one 7-year-old boy. Beyond microdystrophin expression, the company observed reductions in the muscle injury markers CK, AST, ALT, Titin, LDH and eMHC. It describes this as potential stabilization of muscle health.

The company also reports promising signs of muscle function preservation and encouraging early findings in potential cardiac benefit. Heart health is critically important in Duchenne. The company calls its outlook one of cautious optimism.

Global – October 27, 2026 -- Fifty-three boys with Duchenne muscular dystrophy have now received the investigational gene therapy SGT-003, and a Phase 3 trial is already enrolling patients in Australia and Canada. Solid Biosciences shared the update in a letter to the Duchenne community as the annual Parent Project Muscular Dystrophy (PPMD) conference got under way.

The Phase 1/2 trial spans ages 0 to 10

The count comes from the Phase 1/2 INSPIRE DUCHENNE trial and reflects a June 22, 2026, enrollment cutoff. Participants range from 0 to 10 years old. One of them is a 6-month-old boy who, to the company's knowledge, is the youngest child ever dosed with a microdystrophin gene therapy.

Solid reports no cases of four serious safety conditions

The company describes SGT-003 as generally well tolerated. It reports no observed cases of drug-induced liver injury, myocarditis, thrombotic microangiopathy or atypical hemolytic uremic syndrome.

Dallas – October 16, 2026 -- The 21st annual Sam's Night raised $1.7 million, based on preliminary totals at press time, for Parent Project Muscular Dystrophy (PPMD), and donations are still open at SamsNight.org. The Killian family of Rockwall, Texas, hosted the fundraiser on Thursday, October 15, at Victory Social.

Sam's Night has raised more than $10 million since it began

The event started more than two decades ago as Sam's Family Fun Day. It has grown alongside Sam Killian, now 24, who was diagnosed with Duchenne in 2005. Today it is an evening fundraiser that draws the Killians' friends, family, colleagues, and supporters.

The money supports PPMD's work on research, care, advocacy, and access to therapies. PPMD describes itself as the leading U.S. nonprofit driving Duchenne and Becker muscular dystrophy care, research, and advocacy.

Washington – September 08, 2026 -- Parent Project Muscular Dystrophy (PPMD) is joining the global Duchenne and Becker muscular dystrophy community to mark World Duchenne Awareness Day on September 7th, a date recognized by the United Nations.

This Year's Theme Focuses on Access to Care

The 2026 theme, "Access Changes Lives," spotlights the need for people living with Duchenne and Becker to reach trusted information, knowledgeable care, appropriate therapies, clinical trial opportunities, and community support. PPMD says access should not depend on where someone lives or what resources they have.

"Access can change the trajectory of a person's life," said Katherine Beaverson, PPMD's Chief Executive Officer. She added that PPMD is committed to breaking down barriers so families can make informed decisions and benefit from scientific progress.