5 assets in Spinal Muscular Atrophy, Type III

Therapeutic assets addressing Spinal Muscular Atrophy, Type III, rated under Floatz methodology v0.2 and sorted by current rating.

Asset	Sponsor	Phase	Rating
Apitegromab Spinal Muscular Atrophy, Type III	Scholar Rock, Inc.	P3	BB (67)
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) Spinal Muscular Atrophy, Type III	Novartis Gene Therapies	P3	B (60)
Nusinersen Spinal Muscular Atrophy, Type III	Scholar Rock, Inc.	P2	CCC (53)
Risdiplam Spinal Muscular Atrophy, Type III	Scholar Rock, Inc.	P2	CCC (53)
gc101 Spinal Muscular Atrophy, Type III	GeneCradle Inc	P1/2	CC (47)