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rare-disease

A collection of 2 posts
FDA Approves AVLAYAH: First Hunter Syndrome Treatment to Cross Blood-Brain Barrier
rare-disease

FDA Approves AVLAYAH: First Hunter Syndrome Treatment to Cross Blood-Brain Barrier

FDA Approves AVLAYAH: First Hunter Syndrome Treatment to Cross Blood-Brain Barrier Revolutionary therapy overcomes 20-year treatment gap for rare genetic disease The US Food and Drug Administration has granted accelerated approval to AVLAYAH™ (tividenofusp-alfa-eknm), marking a watershed moment in rare disease treatment. This is the first FDA-approved biologic specifically designed
26 Mar 2026 1 min read
Breakthrough Gene Therapy Shows Promise for Children With Muscular Dystrophy
gene-therapy

Breakthrough Gene Therapy Shows Promise for Children With Muscular Dystrophy

Early clinical results demonstrate significant muscle improvement in limb-girdle muscular dystrophy patients
10 Mar 2026 2 min read
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