Sarepta Therapeutics (NasdaqGS:SRPT) reported positive three year functional results from its Phase 3 EMBARK trial of ...

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.

Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...

A mother's love drives her to pursue experimental treatment as two of her four children battle a rare genetic disease that ...

Bedridden and able to move only one finger and one toe, 36-year-old Li Xia from China’s Chongqing region is defying the odds.

US FDA grants Orphan Drug Designation to Atossa Therapeutics’ (Z)-endoxifen to treat Duchenne muscular dystrophy: Seattle Monday, January 19, 2026, 17:00 Hrs [IST] Atossa Therap ...

In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid screening tool to help identify individuals with Duchenne muscular ...

When it comes to children born with rare diseases, early diagnosis can make the difference between life and death. Since many ...

(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...

What the medical literature suggests on who and what might benefit from creatine supplementation and what's less clear.

As a professional opera singer, Sue Lees has performed on countless stages over her decades-long musical career.

Leaders with CureDuchenne say approvals are expected this year for several new treatments for Duchenne MD, making it "an ...