Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single ...

An El Paso mother traveled to Washington, D.C., this week to advocate for her 14-year-old son, who is living with a rare ...

Sarepta Therapeutics CEO Doug Ingram announced Wednesday that he will retire by the end of 2026, citing a “shocking and ...

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the fourth quarter and full year of 2025 and recent business ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking ...

After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales of Duchenne muscular dystrophy gene therapy Elevidys continue to decline.

Last week, Eric Dane passed away from ALS, also known as Lou Gehrig’s disease. He publicly shared his diagnosis in April of ...

Avidity Biosciences, Inc. ("Avidity") , a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCstm) to profoundly improve ...

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

Dual AAV gene therapy using laminin-linking proteins ameliorates muscle and nerve defects in LAMA2-related muscular dystrophy Molecular Therapy (2026), doi: 10.1016/j.ymthe.2026.01.041 ...

The Enquirer and United Way of Greater Cincinnati's 39th annual Wish List campaign raised $148,983 for local families.

On an earnings call, Ingram said he was leaving because his wife and son were recently diagnosed with myotonic dystrophy.