Research
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 […]
Read More ›Biogen Announces New NURTURE Data at the 2019 Annual SMA Conference
Biogen today announced new results from the NURTURE study, adding data to the longest study of spinal muscular atrophy (SMA) in pre-symptomatic infants (n=25). These […]
Read More ›Updated SMA Drug Pipeline Now Available
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 23 active programs, including two approved therapies. 15 pharmaceutical partners. 6 […]
Read More ›Cytokinetics Releases New Data from Reldesemtiv Clinical Study at the 2019 Annual SMA Conference
Cytokinetics, Incorporated, today announced that data from two preclinical studies of reldesemtiv were presented at the 2019 Annual Cure SMA Conference in Anaheim, CA, showing […]
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