Cure SMA would like to thank the Luke 18:1 Foundation for their generous donations to our equipment pool and research programs. The foundation’s generosity will provide support and hope to all those affected by SMA. The Luke 18:1 Foundation was started by Nicole and Daniel Stickane in honor of their son Luke who was diagnosed […]
Cure SMA Receives Generous Funding from Luke 18:1 Foundation Read More »
Cure SMA today announced the launch of our SMA Care Center Network. The SMA Care Center Network is the centerpiece of our efforts to address the changing landscape of SMA. The goal of the SMA Care Center Network is to develop an evidence-based standard of care that will improve the lives of all those affected
SMA Care Center Network and Clinical Data Registry Launched Read More »
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
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
Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA Read More »
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
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
Genentech Releases Community Statement on Risdiplam Read More »
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
Community Spotlight: Adrienne Vollmer Read More »
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
Cure SMA Launches a Family Support App for Your Mobile Device! Read More »
My son Pete and I began advocating to have SMA added to the newborn screening panel in Pennsylvania in December of 2017. Pete was 13-years old, type II SMA. He realized the impact that early diagnosis and treatment was having on SMA babies and thought he could make a difference. He has. Here is our
Community Spotlight: Allyson Henkel Read More »
After Max began receiving Spinraza in 2017, we wanted to do our part to help ensure that children born with SMA would be diagnosed as early as possible. Inspired by many others in the SMA community, we decided to pay a visit to our state senator’s office in Annapolis during SMA Awareness Month to ask
Community Spotlight: The Lasko Family Read More »
