Health Medical Breakthroughs Treatment

Spinal Muscular Atrophy Treatment Research: Experiments in Mice May Help Boost Newly FDA-Approved Therapy for Spinal Muscular Atrophy

Posted on Posted in Spinal Muscular Atrophy News

(SOURCE: JOHNS HOPKINS MEDICINE) – Johns Hopkins researchers along with academic and drug industry investigators say they have identified a new biological target for treating spinal muscular atrophy. They report they have evidence that an experimental medicine aimed at this target works as a “booster” in conjunction with a drug called nusinersen that was recently FDA-approved to […]

Health Medical Breakthroughs Treatment

Spinal Muscular Atrophy Research News: Stanford patient is first infant to receive lifesaving drug for neurodegenerative disease

Posted on Posted in Muscular Dystrophy News, Spinal Muscular Atrophy News

(SOURCE: STANFORD UNIVERSITY MEDICAL CENTER) – Zoe Harting became the first baby in the world to receive an experimental drug that her doctors hoped would save the lives of thousands of infants like her. Zoe has spinal muscular atrophy type 1, a degenerative neuromuscular disease that kills most patients by their second birthday. Before she began receiving […]