Tag: rare disease therapy
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FDA Approves Fasenra as an Under the Skin Shot to Help Reduce Flares in Hypereosinophilic Syndrome
When the FDA approves a new treatment for a rare and difficult condition, it often marks a turning point for patients who have spent years dealing with unpredictable symptoms. That is exactly what happened with the approval of Fasenra, a medication given as an under the skin shot that has now been cleared to help…
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FDA Approves Gene Therapy for Treatment of Spinal Muscular Atrophy — Itvisma (onasemnogene abeparvovec‑brve)
Background and mechanism Spinal muscular atrophy (SMA) is an autosomal‑recessive neurodegenerative disorder caused by loss‑of‑function mutations in the SMN1 gene, resulting in reduced survival motor neuron (SMN) protein, progressive motor neuron loss, and muscle atrophy. Gene‑replacement therapy using adeno‑associated virus (AAV) vectors delivers a functional copy of SMN1 to motor neurons, restoring SMN protein expression…