Research
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
Read More ›Zolgensma Update and Approval Webinars Now Available Online
A recording of last week’s webinar on Zolgensma updates is now available on Cure SMA’s YouTube channel. A presentation of the webinar can be found […]
Read More ›2019 Researcher Meeting Summary: Clinical Research Studies for SMA
The annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year, we had a record setting 735 […]
Read More ›2019 SMA Researcher Meeting Summary: Basic Research Podium Presentations
The annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year, we had a record setting 735 […]
Read More ›2019 SMA Researcher Meeting Summary: Special Session on Spinal and Neuromuscular Circuitry: Exploring Defects in SMA and Potential Therapeutic Targets
The annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year, we had a record setting 735 […]
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