“To see a child dosed with gene therapy at age one and still unable to sit at age five then gain that ability within three months of starting salanersen is unexpected and encouraging,” – Dr. Valeria Sansone, principal investigator of the Phase 1 trial Biogen Inc. announced today that it will advance its investigational […]
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
Cure SMA Awards $50,000 Grant to Tianyuan Shi, PhD, at the University of Hong Kong Read More »
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
We are honored to celebrate the incredible contributions of Dr. Mary Schroth, who is retiring after seven years as our Chief Medical Officer and after many decades of service and care for our community. Honoring Dr. Mary Schroth’s Legacy For several decades, Dr. Mary Schroth has been a driving force in advancing clinical
Cure SMA Celebrates Dr. Mary Schroth as she Retires as Chief Medical Officer Read More »
Make an Impact in Your State and Community Cure SMA is proud to announce the launch of our brand-new State and Local Advocacy Toolkit, now available on the Advocacy webpage! This interactive resource empowers individuals and families in the SMA community to take action and drive change at the local and state level. Whether you’re
New Advocacy Resource: Explore the Cure SMA State and Local Toolkit! Read More »
A drug’s mechanism of action, or MOA, refers to how a drug or other substance produces an effect in the body. Understanding a drug’s mechanism of action (MOA) is crucial for a variety of reasons, including, but not limited to: Improved Understanding: knowing how a drug works can help individuals and families to feel more
Understanding Gene Therapy: A Cure SMA Video Read More »
During our Nunemaker Family Research Community Challenge, all donations will be matched up to $150,000! Cure SMA is proud to announce the launch of our Nunemaker Family Research Community Challenge, an incredible opportunity to double your impact in our efforts to support spinal muscular atrophy (SMA) research. Thanks to the generosity of the Nunemaker Family,
Double Your Power, Double Our Progress: Help Cure SMA Unlock $150,000 for SMA Research! Read More »
Since 2017, the Cure SMA Community Update Survey has collected data and information on the SMA community’s experiences and unmet needs. Every single individual with SMA and their families bring a unique perspective that, collectively, helps us adapt to the changing landscape of SMA. Data from the survey also informs Cure SMA’s advocacy agenda and
Last Chance to Participate in the Annual Cure SMA Community Update Survey Read More »
Novartis Gene Therapies recently released an update on their continued collaboration with the SMA community through events, advisory boards, clinical insights, and ongoing efforts to center patient, family, and community voices in their work. Read the full community update letter here. “As winter turns to spring, we want to recognize that we have had an
Novartis Gene Therapies Releases Spring SMA Community Update Letter Read More »
