Pyridostigmine

Spinal Muscular Atrophy, Type III

UMC UtrechtSmall moleculePhase 2Dormant

62/ 100

Provisional rating · Methodology v0.2

Rating breakdown

5 Active assets in the Spinal Muscular Atrophy, Type III cohort.

Asset / Sponsor	Phase	Rating
Apitegromab Scholar Rock, Inc.	Phase 3	BB
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 Gene Therapies	Phase 3	B
Olesoxime, E- Hoffmann-La Roche	Phase 2	B
Risdiplam Scholar Rock, Inc.	Phase 2	CCC
Nusinersen Scholar Rock, Inc.	Phase 2	CCC
AMIFAMPRIDINE Catalyst Pharmaceuticals, Inc.	Phase 2	CCC
Sodium Phenylbutyrate Westat	Phase 1/2	CCC
gc101 GeneCradle Inc	Phase 1/2	CC