FDA Approves First Drug for Hunter Syndrome Brain Complications in Children
Avlayah becomes the first treatment targeting neurologic symptoms of rare genetic disorder affecting 500 Americans.
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Avlayah becomes the first treatment targeting neurologic symptoms of rare genetic disorder affecting 500 Americans.
Avlayah (tividenofusp alfa-eknm) becomes the first therapy approved to address brain damage in Hunter syndrome, a rare pediatric disorder.
Two breakthrough approvals target Hunter syndrome brain complications and cerebral folate deficiency for the first time.
Wellcovorin becomes the first approved treatment for cerebral folate deficiency tied to FOLR1 gene variants, offering hope for a rare neurological condition.
A roundup of recent FDA brain-health approvals including lecanemab's full Alzheimer's approval and a new glioma drug.
From Alzheimer's to Parkinson's, a wave of novel FDA approvals is expanding treatment options for serious neurological conditions.
FDA approves an updated Spinraza regimen for spinal muscular atrophy, targeting improved CSF drug levels for better neurological outcomes.