Spinal Muscular Atrophy

Scholar Rock Holding Corp (NASDAQ: SRRK) will showcase data from an exploratory analysis of the ambulatory cohort in the Phase 2 TOPAZ trial of apitegromab in individuals with later-onset spinal muscular atrophy (SMA). The TOPAZ study assessed the safety, tolerability, PK/PD, and efficacy of...

Scholar Rock Holding Corp (NASDAQ: SRRK) announced the study design for the SAPPHIRE Phase 3 trial of apitegromab, a selective inhibitor of the activation of latent myostatin. The trial will evaluate the efficacy and safety of apitegromab in patients with non-ambulatory Type 2 and...

Roche Holding AG (OTC: RHHBY) has highlighted new data for its oral spinal muscular atrophy (SMA) treatment Evrysdi, showing its benefits when given to pre-symptomatic babies with the rare disease. The data were presented at the World Muscle Society (WMS) Virtual Congress. The...

Biogen Inc's (NASDAQ: BIIB) Spinraza (nusinersen), spinal muscular atrophy (SMA) drug launched in early 2017, faces new competition from Roche Holdings AG's (OTC: RHHBY) oral entrant Evrysdi (risdiplam).  Biogen has unveiled plans to test Spinraza in...

Novartis AG (NYSE: NVS) announced new data from the completed two-copy cohort of Phase 3 SPR1NT trial of Zolgensma (onasemnogene abeparvovec), gene therapy for spinal muscular atrophy (SMA). The data were presented at the European Academy for Neurology Virtual Congress 2021. In the...

Scholar Rock (NASDAQ: SRRK) shares dropped despite the company reporting positive 12-month data on spinal muscular atrophy (SMA) candidate apitegromab. The 12-month update was broadly in line with the six-month data that was announced in October last year. Most effects...

Scholar Rock (NASDAQ: SRRK) has announced top-line data from the TOPAZ Phase 2 trial evaluating apitegromab (SRK-015) in patients with Type 2 and Type 3 spinal muscular atrophy (SMA). In Cohort 1 (pooled population), the mean change from baseline in Revised...

Data presented by Cytokinetics, Inc. (NASDAQ: CYTK) showed CK-107 has the ability to positively impact two different animal models of spinal muscular atrophy. Rodman & Renshaw’s Joseph Pantginis reiterated a Buy rating on the company, with a price target of $25. Positive Indications The...

Citron Research's latest target is AveXis Inc (NASDAQ: AVXS), a clinical-stage gene therapy company that focuses on creating gene therapy treatments for patients suffering from neurological genetic diseases. Citron Research, the notable short-selling research firm, blasted AveXis in a report dated...