Tears gathered in Jeremy Bray’s eyes as he absorbed the news that his pleas for Manitoba’s government to cover his life-sustaining treatment hadn’t changed the health minister’s mind. Bray, 30, who ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared progress in the Phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 ...

Shares of BridgeBio Pharma rose after the company reported positive data in the phase three study of its treatment of limb-girdle muscular dystrophy. The stock rose 10% to $59.74 in premarket trading ...

Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Satellos Bioscience ( (TSE:MSCL)) has ...

Duchenne muscular dystrophy (DMD) causes muscle weakness that becomes more severe over time. While there is no cure, treatments can help slow the progression and manage symptoms and complications.

People with Duchenne muscular dystrophy have a shorter-than-average life expectancy due to changes in muscles that affect breathing, heart activity, and other functions. However, treatment advances ...

SAR446268 is a one-time adeno-associated viral gene therapy that delivers a vectorized RNA interference to silence DMPK expression. The Food and Drug Administration (FDA) has granted Fast Track ...

There is no cure for DMD, but treatments can slow the progression of the disease and help improve quality of life. Duchenne muscular dystrophy (DMD) is a chronic condition that causes a gradual loss ...

LONDON, Sept. 15, 2025 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held clinical-stage specialty biopharmaceutical company focusing on rare genetic disorders with limited or no ...

AMO-01 is being investigated for treatment of Phelan-McDermid syndrome and AMO-04 as a clinic-ready potential medicine for Rett syndrome and related disorders. AMO-02, AMO-01 and AMO-04 are ...