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Scholar Rock to Present Preclinical and Phase 1 Clinical Data on SRK-015 at the World Muscle Society Congress
Scholar Rock recently announced that a poster presentation highlighting preclinical and Phase 1 healthy volunteer data for SRK-015 will be presented at the World Muscle Society Congress. The 24th Annual […]
Read More ›Landmark NURTURE Study of Infants with Spinal Muscular Atrophy (SMA) Treated Pre-Symptomatically with Spinraza Published in Neuromuscular Disorders
Biogen today announced that the journal Neuromuscular Disorders has published data from NURTURE, the first study investigating a treatment targeting the underlying cause of spinal muscular atrophy (SMA) in infants […]
Read More ›Risdiplam Data Presented at World Muscle Society Congress Demonstrates Continued Benefit for Patients with Spinal Muscular Atrophy
Today, new data was presented demonstrating the ongoing benefit of risdiplam (RG7916) for the treatment of all types of spinal muscular atrophy (SMA). The data was presented at the 24th […]
Read More ›Community Spotlight: Paul Guarino of PG Sports
Paul Guarino was ahead of his time when he started PG Sports as a Twitter page in 2011. Initially, the West Haven, CT native created a simple logo and shirts […]
Read More ›AveXis Presents New Data at EPNS Continuing to Show Significant Therapeutic Benefit of Zolgensma® in Prolonging Event-free Survival Now up to 5 Years of Age in Patients with SMA Type 1
AveXis, a Novartis company, recently announced that new interim data from the Phase 3 SPR1NT trial in pre-symptomatic patients as well as interim data from the ongoing Phase 3 STR1VE […]
Read More ›Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study, DEVOTE, Evaluating a Higher Dose of SPINRAZA and Additional Data in a Broad Range of Patients
Biogen today announced updates to the SPINRAZA (nusinersen) clinical development program including the initiation of a new global clinical trial, DEVOTE. The DEVOTE study will evaluate if a higher dose […]
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