New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ...

目前，虽然基因编辑疗法为 DMD 的治疗带来了一丝希望，但临床应用的道路上仍布满荆棘，比如难以确定临床疗效、无法有效控制潜在的脱靶效应等。而糖皮质激素（如地夫可特、泼尼松）虽能延缓疾病进展，却也伴随着肥胖、行为改变、身材矮小和骨质减少等诸多副作用，就像 “饮鸩止渴”，让患者陷入两难境地。因此，寻找更安全有效的治疗方法，成为了医学领域亟待攻克的难题。

Having found that in biomedical research, the Bird Rock resident and scientist at La Jolla’s Sanford Burnham Prebys has ...

Dominic Olsen is a bright and determined 10-year-old boy living with Duchenne Muscular Dystrophy, a rare disease that has ...

Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases where ...

Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...

Dyne Therapeutics (DYN) announced new long-term clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 demonstrating unprecedented ...

A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular ...

Roland Beres sits down with April Gannon of the Muscular Dystrophy Association to discuss the importance of raising money and ...

Peptris Technologies out-licenses India’s first AI discovered drug candidate to Revio Therapeutics: Our Bureau, Bengaluru Monday, March 17, 2025, 12:50 Hrs [IST] Peptris Technol ...