AveXis has announced that they have completed enrollment in a Phase 1 study testing systemic delivery of gene therapy for SMA. The trial has enrolled a total of 15 patients who met enrollment criteria of diagnosis of SMA Type 1 before six months of age, with two copies of the SMN2 backup gene, as determined
AveXis Announces the Completion of Enrollment for Phase 1 Gene Therapy Trial Read More »
Cytokinetics has opened enrollment for a Phase 2 study testing CK-2127107 in teens and adults with SMA type II, III or IV. We are excited to see the continued progress of this drug into the next phase of clinical trials, and particularly excited to see this trial focused on teens and adults. This latest trial
Cytokinetics Opens Enrollment for Phase 2 Trial Read More »
An important goal of our research funding strategy is to share scientific findings with the broader scientific community. To help accomplish this goal, scientists who receive Cure SMA funding often publish their findings in peer-reviewed journals. Published articles allow the experiments and results to be reviewed and vetted by other scientists, who may then use
Cure SMA Funding Leads to 18 New Research Publications in 2015 Read More »
Our approach to research is focused on continually expanding the pipeline of potential drugs and therapies for SMA, cultivating new approaches and advancing promising ideas. Every year, the pipeline gets broader and deeper. We’ve seen enormous growth over the past decade, and we expect that growth to continue in the coming years as more projects
Cure SMA Funds 28 Research Projects in 2015, as Drug Pipeline Grows to 18 Programs Read More »
Each year, Cure SMA provides thousands of families with vital support and resources to help them live active, engaged and hopeful lives. Our programs provide tangible support for families affected by SMA, easing the difficulties of an SMA diagnosis, and showing each family that they are not alone and that there is hope. Our family
Cure SMA Reaches Thousands of Families in 2015 Read More »
On December 1, more than 120 people from the SMA community—including government, and industry partners—gathered for the 5th Annual “Hope on the Hill” Congressional Dinner. Several Members of Congress attended the dinner, including Speaker of the House Paul Ryan, Congressman Sean Duffy, Congressman Richard Hudson, Congressman Bill Huizenga, Congressman Erik Paulsen, Congressman David Schweikert, and
5th Annual Congressional Dinner Brings Together Families, Government and Industry Read More »
ELK GROVE VILLAGE, Ill. and SOUTH SAN FRANCISCO, Calif., Dec 8, 2015 – Cure SMA and Cytokinetics Inc. (Nasdaq: CYTK), a leading muscle biology company, today announced an expanded partnership to increase education, awareness and fundraising for spinal muscular atrophy (SMA). As a National Gold Partner, Cytokinetics will lend support to key national and local
Members of Cure SMA’s Scientific Advisory Board (SAB) have gathered in Chicago, meeting to advance Cure SMA’s research goals. Our SAB is made up of highly respected SMA researchers, including experts in child neuromuscular disorders, SMA biology, SMN splicing, motor neurons, mouse and other animal models, and more. The SAB focuses on identifying the most
Cure SMA Scientific Advisory Board Meets in Chicago Read More »
In the last few years, we’ve gotten closer to an approved treatment than we ever have been. Through research funded by Cure SMA, we’ve identified the cause of SMA, and are developing multiple ways to treat SMA. But with this great progress comes even greater needs. That’s why we’ve set a goal of raising $750,000
Great Progress, Greater Need Read More »
The 5th Annual South Florida Chapter Gala of Hope was a fantastic evening to raise funds and awareness of SMA. This year’s event has already raised over $224,000, and money is still being tabulated! In its five-year history, the event has raised an incredible total of over $700,000 for SMA research and family support. The
5th Annual South Florida Gala of Hope Raises Record Total of $224,000 Read More »
