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Novartis Gene Therapies — Rare Disease Pipeline | Kisho | Kisho

Novartis Gene Therapies

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FDA Approvals

Rare Diseases

News Articles

Disease	Designation	Approved
X-linked distal spinal muscular atrophy type 3	-	FDA Approved
adult progressive spinal muscular atrophy, Aran Duchenne type	-	FDA Approved
adult-onset proximal spinal muscular atrophy, autosomal dominant	-	FDA Approved
arthrogryposis spinal muscular atrophy	-	FDA Approved
autosomal dominant childhood-onset proximal spinal muscular atrophy	-	FDA Approved
autosomal dominant childhood-onset proximal spinal muscular atrophy with contractures	-	FDA Approved
autosomal dominant childhood-onset proximal spinal muscular atrophy without contractures	-	FDA Approved
autosomal recessive distal spinal muscular atrophy 1	-	FDA Approved
autosomal recessive distal spinal muscular atrophy 2	-	FDA Approved
bulbospinal muscular atrophy	-	FDA Approved
congenital benign spinal muscular atrophy dominant	-	FDA Approved
early-onset progressive encephalopathy-spastic ataxia-distal spinal muscular atrophy syndrome	-	FDA Approved
infantile-onset X-linked spinal muscular atrophy	-	FDA Approved
prenatal-onset spinal muscular atrophy with congenital bone fractures	-	FDA Approved
proximal spinal muscular atrophy	-	FDA Approved
scapuloperoneal spinal muscular atrophy, autosomal dominant	-	FDA Approved
scapuloperoneal spinal muscular atrophy, autosomal recessive	-	FDA Approved
spinal muscular atrophy	-	FDA Approved
spinal muscular atrophy type 0	-	FDA Approved
spinal muscular atrophy with congenital bone fractures 1	-	FDA Approved
spinal muscular atrophy with congenital bone fractures 2	-	FDA Approved
spinal muscular atrophy with intellectual disability	-	FDA Approved
spinal muscular atrophy with microcephaly and mental subnormality	-	FDA Approved
spinal muscular atrophy with respiratory distress type 2	-	FDA Approved
spinal muscular atrophy, Ryukyuan type	-	FDA Approved
spinal muscular atrophy, distal, autosomal recessive, 6	-	FDA Approved
spinal muscular atrophy, facioscapulohumeral type	-	FDA Approved
spinal muscular atrophy, infantile, James type	-	FDA Approved
spinal muscular atrophy, lower extremity-predominant, 2b, prenatal onset, autosomal dominant	-	FDA Approved
spinal muscular atrophy, segmental	-	FDA Approved
spinal muscular atrophy, type 1	-	FDA Approved
spinal muscular atrophy, type II	-	FDA Approved
spinal muscular atrophy, type III	-	FDA Approved
spinal muscular atrophy, type IV	-	FDA Approved
spinal muscular atrophy-Dandy-Walker malformation-cataracts syndrome	-	FDA Approved
spinal muscular atrophy-progressive myoclonic epilepsy syndrome	-	FDA Approved

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