On Thursday, March 7, Cure SMA testified on behalf of the SMA community at a meeting of the Institute for Clinical and Economic Review (ICER). ICER is an independent health policy group that evaluates the effectiveness of drug treatments, and the costs associated with those treatments. The purpose of the meeting was to review both […]
Cure SMA Advocates for SMA Community at ICER Hearing Read More »
Dear members of the SMA community, As we celebrate Rare Disease Day 2019, we are honored to continue supporting the spinal muscular atrophy (SMA) community. In recent weeks and months, we engaged the community on a number of different initiatives and are excited to share updates on our activities, support of new clinical research and
Biogen Provides 2019 Community Update on Spinraza Read More »
On Monday, March 11th, at 12pm CST (10am PST/11am MST/1pm EST) Genentech/Roche and Cure SMA will co-host a webinar for the SMA community. During the webinar, you’ll hear from Genentech/Roche on their history working with the SMA community, and their investigational medicine risdiplam. You’ll hear updates on their clinical trials in pre-symptomatic & Types 1,
Genentech/Roche and Cure SMA to Co-Host Webinar Read More »
Cure SMA is pleased to announce that we will be continuing the Summit of Strength Program in 2019, adding 20 new events from March through November. The Summit of Strength is a single-day educational event hosted by Cure SMA. Summits are crafted to provide information for people of all ages and types of SMA, and
New Dates and Locations Announced for 2019 Summits of Strength Read More »
Scholar Rock today announced positive interim results from the ongoing Phase 1 single- and multiple-ascending dose clinical trial of SRK-015 in healthy adult volunteers. SRK-015, a highly specific inhibitor of myostatin activation, was observed to be well tolerated with no apparent safety signals. By demonstrating robust and sustained target engagement in humans, pharmacologic effects on
The SMA community has been lobbying state and federal government for years. Kristen Smith shares how she became involved with advocacy. Kristen Smith describes herself as an artist and advocate. A powerful combination during a time where people across the country are standing up for their community and speaking out for their rights. Kristen is
Community Spotlight: Kristen Smith Read More »
We are thrilled to share that both Arkansas and Florida recently adopted SMA to their state newborn screening panels! They now join the growing list of states that have adopted SMA to their newborn screening panels. Arkansas Adopts SMA Newborn Screening Congratulations to Arkansas, who just passed legislation to add SMA to their newborn screening
Arkansas and Florida Adopt SMA Newborn Screening Read More »
Audrey Lewis founded Families of SMA, now Cure SMA, 34 years ago. Audrey recognized early on the importance of attracting new and talented researchers to SMA, with the hope that they would commit their careers to developing a treatment and cure for SMA. Cure SMA honors Audrey’s legacy with the Audrey Lewis Young Investigator Award,
Amber Bosselman is your typical 22-year-old college senior. She is currently studying psychology in Idaho and planning to get her master’s degree in industrial psychology. Amber’s life is similar to other students, except she was born with a rare disease called spinal muscular atrophy (SMA). SMA is a disease that robs people of strength by
Community Spotlight: Amber Bosselman Read More »
1 in 20 people will live with a rare disease at some point in their life. Rare Disease Day improves knowledge amongst the general public of rare diseases while encouraging researchers and decision makers to address the needs of those living with rare diseases. Throughout February, show your support of everyone affected by SMA and
Show Your Rare. Show You Care. Read More »
