We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers a session on newborn screening. The session was moderated by Kathryn Swoboda, MD. Newborn Screening for SMA: Ethics, Rationale, and Implementation Data generated in mouse models of […]
2015 SMA Researcher Meeting Summary: Newborn Screening Read More »
At the Annual SMA Conference, representatives from six of the seven programs currently in clinical trials gave an update on their programs. Of these six programs, four—gene therapy, ISIS-SMNRx, RG7800, and LMI070—treat the underlying genetics of SMA. Gene therapy aims to correct the mutation in the SMN1 gene that causes SMA, and the other three
Updates from Drug Programs in Clinical Trials Read More »
We will be posting a series of summaries from our 2015 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This update covers the special session, “Emerging Trends in Motor Neuron Pathobiology.” This summary was written by Cure SMA Scientific Advisory Board member Doug Kerr, MD, PhD. Emerging Trends in
2015 SMA Researcher Meeting Summary: Emerging Trends in Motor Neuron Pathobiology Read More »
The House of Representatives has passed 21st Century Cures. Cure SMA is proud to support this legislation. Thank you for your calls and tweets to voice your support to your Representatives. They really made a difference! So what happens next? The Senate is busy drafting their version of the bill, called “Innovation,” and we expect
House of Representatives Passes 21st Century Cures Read More »
Each year, while much of our community is gathered together, we use the Annual SMA Conference to recognize several individuals, families and foundations who have provided special support to the Annual SMA Conference and the SMA community. Here are this year’s awardees. On behalf of the entire community, thank you for all you do! Shaina
Seven Individuals and Families Recognized for Their Contributions to the Community Read More »
At last week’s Annual SMA Conference, Cytokinetics released additional data from their Phase 1 study of CK-2127107, a skeletal muscle activator. Cytokinetics, in partnership with Astellas, has already announced that a Phase 2 study will begin later this year. Our partnership with Cytokinetics highlights two strengths of Cure SMA’s research model: providing seed funding to
Cytokinetics Releases Update on Phase 1 Trial at Annual SMA Conference Read More »
At last week’s Annual SMA Conference, Isis Pharmaceuticals, Inc. provided an update on children with spinal muscular atrophy (SMA) who have completed the open-label, Phase 2 multiple-dose study of ISIS-SMNRx and are continuing to receive treatment in an open-label extension (OLE) study. Consistent with earlier observations, increases in muscle function scores and additional motor function
Isis Announces Additional Trial Results at Annual SMA Conference Read More »
Cure SMA has announced plans to fund $1.995 million in new research grants over the next several months. This includes funding for all four areas of our research model—basic research, drug discovery, clinical trials, and clinical care research—plus funding for the 2016 SMA Researcher Meeting, held each year as part of the Annual SMA Conference.
Cure SMA Announces Nearly $2 Million in New Planned Research Funding Read More »
We are very excited to announce that Dr. Mary Schroth has joined us as our medical professional education consultant. She will be working with us a few days each month, depending on her clinic schedule, as Mary remains deeply committed to working with her patients. Mary will also continue to chair our Medical Advisory Council.
Dr. Mary Schroth Joins Cure SMA as Medical Professional Education Consultant Read More »
The latest issue of Compass, our research publication, is now available online. This summer 2015 issue includes information on our recent drug discovery grants, plus an update to our SMA drug pipeline. Developing a treatment and cure for SMA is the driving force behind Cure SMA, and the goal of our three-stage research model. Drug
Read the Summer 2015 Issue of Compass Read More »
