—Navigating the intricacies of health insurance policies to get coverage for needed medical care is often a huge burden for patients and caregivers battling a rare disease. A new study opens a window ...
Cure Rare Disease (CRD), a 501(c)(3) nonprofit biotechnology company developing genetic therapies for ultra-rare neuromuscular diseases, today announced that the U.S. Food and Drug Administration (FDA ...
Partnership leverages AI-powered genome design to optimize AAV constructs, accelerate lead candidate selection, and improve drug substance manufacturability for rare and ultra-rare neuromuscular ...
In some cases, an inability to balance is referred to as a symptom known as ataxia. But ataxia can also be its own condition ...
AUSTIN (KXAN) — Most parents walk through life expecting and hoping to be there for every significant milestone in their children’s lives. Graduating from high school and college, little league games, ...
The state House of Representatives Health Committee has approved legislation adding Duchenne muscular dystrophy and Gaucher ...
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FDA Gives Broad Approval to Gene Therapy for Rare Neurologic Disease
The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in adults and pediatric patients 2 years and older with a confirmed mutation in ...
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