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Risdiplam

Spinal Muscular Atrophy

Scholar Rock, Inc.Small moleculePhase 4 / ApprovedActive
A
80/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
70/ 100
Competitive Position
95/ 100

Competitive context

19 Active assets in the Spinal Muscular Atrophy cohort.

Asset / SponsorPhaseRating
Nusinersen
Scholar Rock, Inc.
Phase 4A
oav101
Novartis Pharmaceuticals
Phase 4BBB
Non-Replicating Recombinant Self-Complementary (Sc) Adeno-Associated Virus Serotype 9 (Aav9) Vector Containing The Cdna Of The Survival Of Motor Neuron 2 (Smn2) Gene Under The Control Of The Hybrid Cytomegalovirus (Cmv) Enhancer/Chicken Beta-Actin Promoter (Cba)
Novartis Pharmaceuticals
Phase 4BBB
Apitegromab
Scholar Rock, Inc.
Phase 3BB
Valproic Acid
All India Institute of Medical Sciences
Phase 3BB

View all 38 →

Same molecule, other indications

Risdiplam is also rated in:

spinal muscular atrophy, type IIActive
CCC
Indicative · Phase 2
53 / 100
View asset →
spinal muscular atrophy, type IIIActive
CCC
Indicative · Phase 2
53 / 100
View asset →
neuromuscular diseaseActive
CCC
Indicative · Phase 2
53 / 100
View asset →

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