Parent Project Muscular Dystrophy, Foundation to Eradicate Duchenne Announce 2026 Advocacy Leadership Awards Recipients Honoring Bipartisan Champions in Congress

Parent Project Muscular Dystrophy (PPMD), in partnership with the Foundation to Eradicate Duchenne (FED), today announced Representative Troy Balderson (OH-12), Senator Susan Collins (ME), Senator Amy ...

ITF Therapeutics LLC Presents New Data and Analyses on Givinostat for Treatment of Duchenne Muscular Dystrophy at 2026 MDA Clinical and Scientific Conference

ITF Therapeutics LLC, the U.S. rare disease affiliate of Italfarmaco, today announced the presentation of ten abstracts at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference ...

Benitec Biopharma Announces Positive Interim Phase 1b/2a Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 Treatment at the 2026 Muscular ...

Patients treated with low dose BB-301 and high dose BB-301 experienced significant improvements in throat closure, throat emptying, and total dysphagic symptom burden - OPMD Patients treated with low ...

Dyne Therapeutics Rises on Positive Data for Duchenne Muscular Dystrophy Treatment

Shares of Dyne Therapeutics gained after the company shared positive data from a continuing trial of a potential Duchenne muscular dystrophy treatment. The stock rose 14%, to $16.93, midday Monday.

Firetruck pull raises funds and awareness for boy battling muscular dystrophy

A test of strength that raises money for a good cause. Sunday was New Bern Fire and Rescue’s annual firetruck pull.Teams of ...
KOBI-TV NBC5 / KOTI-TV NBC2

Part 2: Local boy with Duchenne Muscular Dystrophy

Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...

Tenaya Therapeutics Presents Preclinical Data at MDA 2026 Highlighting TN-301’s Potential to Correct Skeletal and Cardiac Muscle Decline in Duchenne Muscular Dystrophy

Tenaya’s Highly Selective HDAC6 Inhibitor TN-301 Outperformed Approved Pan-HDAC Inhibitor Givinostat in Improving Muscle Function and Correcting ...

First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

Catalyst Pharmaceuticals to Showcase Real‑World Findings in Duchenne Muscular Dystrophy and Host Vamorolone-Focused Symposium at the 2026 MDA Clinical & Scientific Conference

The MDA meeting is a key opportunity to highlight the expanding real‑world evidence shaping care for the Duchenne community,” said Will Andrews, MD, Chief Medical Officer of Catalyst. “Through our ...
STAT

Sarepta Therapeutics lays off one-third of workforce in drastic cost-cutting move

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
TMCnet

Precision BioSciences Receives FDA Fast Track Designation for PBGENE-DMD and Announces Duchenne Muscular Dystrophy Investor Event

"Fast Track designation is an important regulatory milestone for PBGENE-DMD and reflects the significant unmet need in DMD," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "We ...

Brogidirsen (NS-089/NCNP-02) 4.5-Year Clinical Trial Data for the Treatment of Duchenne Muscular Dystrophy Presented at 2026 MDA Clinical & Scientific Conference

NS Pharma, Inc. (NS Pharma, New Jersey, USA; President, Yukiteru Sugiyama ), a biopharmaceutical leader in rare diseases and subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today ...