Author name: Morgan Tuttle

New Spinraza Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with SMA

Biogen, Inc. announced new interim results from NURTURE, an ongoing open-label, single-arm efficacy and safety study of SPINRAZA® (nusinersen) in 25 presymptomatic infants with SMA. The data were presented Sunday in a late-breaking session at the 23rd Annual Congress of the World Muscle Society (WMS) held in Mendoza, Argentina. The interim analysis evaluated survival and […]

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Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA

Cytokinetics, Incorporated and Astellas Pharma Inc. today provided an update for their collaborative skeletal muscle program, including clinical trial updates for reldesemtiv in SMA. In June, Cytokinetics announced data from a Phase 2 double-blind, randomized, placebo-controlled clinical study in patients with SMA which was designed to determine potential pharmacodynamic effects of a suspension formulation of

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Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam (RG7916) in spinal muscular atrophy (SMA). In the FIREFISH study in Type 1 SMA, six out of 14 infants (43 percent) were able to sit

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Genentech Releases Community Statement on Risdiplam

The following statement is a community update from Genentech/Roche on the risdiplam studies FIREFISH and SUNFISH. Dear SMA community, We are happy to share an update on our FIREFISH and SUNFISH studies of risdiplam. We would also like to acknowledge the tremendous support and partnership of SMA Patient Groups around the world and to thank

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Community Spotlight: Adrienne Vollmer

In May, Governor Eric Holcomb held a signing ceremony for Indiana House Bill 1017, adopting newborn screening for SMA and SCID. Dubbed “Graham’s Bill” in honor of Graham Vollmer, the bill was largely advanced by the efforts of Adrienne Vollmer, newborn screening advocate and SMA mom. The signing was held at the Cure SMA Walk

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Cure SMA Launches a Family Support App for Your Mobile Device!

Cure SMA is excited to announce the launch of a new mobile app for the SMA community. The Cure SMA Guide app is a family support program that takes a new approach to provide useful tools and information related to SMA care, to use at home and on-the-go. The app is now available to download

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Cure SMA’s Approach to Newborn Screening

The complexities of newborn screening require a sophisticated, multifaceted approach. With this in mind, Cure SMA has been working on newborn screening through several different avenues, bringing our full resources to ensure that babies born with SMA have the opportunity to receive early treatment for maximum effectiveness. Advocacy We are working with family advocates, clinicians,

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