The @fda.gov has approved the first gene therapy for children with Sanfilippo syndrome type A.

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#ClinicalMondays #GeneTherapy #SanfilippoSyndrome #Pediatrics #OrphanDrug #FastTrack #BreakthroughTherapy

The @fda.gov has approved the first therapy for glycogen storage disease type Ia (GSDIa).

Genglycos (pariglasgene brecaparvovec-opnr) received accelerated approval for patients aged 8 years and older.

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#ClinicalMondays #GeneTherapy #RareDisease #GSDIa #FDA

Merck and Moderna have announced positive top-line results from the Phase 3 INTerpath-001 trial of intismeran autogene (V940) + pembrolizumab in patients with resected high-risk melanoma.

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#ClinicalMondays #Melanoma #CancerVaccine #mRNA #Immunotherapy