Union Minister H D Kumaraswamy has assured assistance for a two-year-old child suffering from a rare genetic disorder after ...

Outperformed Approved Pan-HDAC Inhibitor Givinostat in Improving Muscle Function and Correcting Drivers of DMD Cardiomyopathy New Data Confirm TN-301’s Differentiated Mechanism and Opportunities to ...

Atamyo Therapeutics, a biotechnology company specializing in the development of next-generation gene therapies for limb-girdle muscular dystrophy (LGMD), announced at the MDA (Muscular Dystrophy ...

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 ...

After a heart attack, the heart struggles to recoup and maintain energy. One-third of patients develop heart failure as a ...

Early diagnosis of rare diseases in children can significantly improve treatment outcomes and quality of life. Learn symptoms, challenges, and why early detection is crucial for managing pediatric ...

Pratteln, Switzerland, February 27, 2026 – Santhera Pharmaceuticals (SIX: SANN) today announces that the Spanish Interministerial Commission on Prices of Medicines, has proposed to include AGAMREE® ...

Dyne announced the results of new analyses of cardiac and pulmonary function amongst all DELIVER participants who were randomized to z-rostudirsen treatment at baseline (any dose 1) and for whom ...

Dyne Therapeutics maintains a strong $1.1B cash position, funding operations into mid-2028 despite heavy R&D and operating ...

As policies promise support and courts intervene, thousands of rare disease patients remain trapped between soaring drug ...

When closely related species mate, their offspring sometimes survive but cannot reproduce. This pattern often affects males ...

Experts call for early diagnosis, prophylaxis in hemophilia, improved MS policy frameworks, genomic testing access and ...