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 ...

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 ...

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 ...

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 ...

Bureaucracy's Rot Runs Amok at FDA ...

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

HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 -- 48-week trial will enroll approximately 150 ...

Vinay Prasad’s exit — his second from the agency in less than a year — comes after sharp criticism of his handling of drug ...

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

New analyses out to 24-months showed improvement in heart and lung function compared to expected declines in DMD natural history - - Data expand on previously reported results demonstrating that z-ros ...