World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD
A Life-Changing Gene Therapy for Severe Aromatic L-Amino Acid Decarboxylase Deficiency.
A one-time gene therapy for the treatment of adults with hemophilia B
A personalized gene therapy to treat boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD)
Gene therapy for beta-thalassemia using patient’s own cells with beta-globin gene.
ZOLGENSMA is a prescribed gene therapy indicated for the treatment of spinal muscular atrophy (SMA) in children under the age of 2.
Treating high-risk neuroblastoma in children (≥1 year) and adults with relapsed or refractory cases.
Prescribed for HLA-A*02:01–positive adults with unresectable or metastatic uveal melanoma, an aggressive form of eye cancer.
Prescribed medication that replenishes the vital hormone leptin, addressing its deficiency in individuals with generalized lipodystrophy (GL).
Luxturna is a medicine that is used to treat inherited retinal dystrophy, a rare genetic disorder.
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