Latest News
Cure SMA Partners with the Clements Twins and Nail & Bone
The Clements Twins, Ava & Leah, are raising money and awareness for spinal muscular atrophy (SMA) through a partnership with Nail & Bone, a socially conscious nail polish company. Their […]
Read More ›AveXis Presents Updated STRONG Data Demonstrating a Higher Mean Increase in Hammersmith Functional Motor Scale-Expanded (HFMSE) Scores Among Older SMA Type 2 Patients Following One-time Intrathecal Administration of AVXS-101
AveXis, a Novartis company, announced on October 5, 2019 new interim data from the Phase 1/2 STRONG study for intrathecal (IT) administration of AVXS-101, demonstrating older patients (≥ 2 years […]
Read More ›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 […]
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