Novartis has published a paper in the journal Nature Chemical Biology, detailing promising results in their program to develop an orally available SMA drug that corrects SMN2 splicing. In testing, the drug increased SMN protein levels and extended survival in a severe mouse model of SMA. Novartis is currently testing this drug in a Phase […]
Novartis Launches Phase 2 Clinical Trial Read More »
Cure SMA has announced a new partnership with the District Policy Group, a boutique lobbying, public policy, and advocacy practice, to advocate for several key policy areas on behalf of the SMA community. With seven drug programs now in clinical trials, the Patient-Focused Drug Development emphasis of PDUFA V, and the progress toward passage of
Cure SMA Announces Partnership with The District Policy Group Read More »
The 15th Annual New England Chapter Walk-n-Roll was held on Saturday, May 16, at beautiful DCR Wompatuck Park in Hingham, MA. The sun was shining as more than 600 people participated in the 1.5 mile walk. Last year, this event hit a milestone by reaching $1 million raised in its history, and this year has
15th Annual New England Walk-n-Roll Raises Over $100,000 Read More »
For the seventh consecutive year, Cure SMA has received a grant from the National Institutes of Health (NIH) for the SMA Researcher Meeting. This year’s grant will go toward conference expenses for fellows, who are younger researchers in training. With the help of this NIH grant, Cure SMA generously subsidizes the cost of registration and
Cure SMA Receives NIH Grant Read More »
Cure SMA mourns the loss of Ken Emerson, volunteer at the Cure SMA National Office. Since his grandson Ryan was diagnosed with SMA type III, Ken became everyone’s favorite visitor at the National Office. Ken even “earned” his own chair at the office, which he was proud to claim as his own! He always put
In Memory of Ken Emerson Read More »
Yesterday, the 21st Century Cures bill was unanimously passed by the House Energy and Commerce Committee. 21st Century Cures would overhaul how the Food and Drug Administration evaluates new medical products and provide $10 billion in mandatory funding for the National Institutes of Health. Passage by the Committee is a significant milestone for this important
21st Century Cures Unanimously Passes Committee Read More »
Much as families in our community often work together to start a chapter, organize an event, or raise awareness of SMA, researchers must work together in order to find the fastest and most effective path to a cure for SMA. In addition to providing funding for ongoing SMA research, Cure SMA provides several opportunities for
Collaborative Ways Cure SMA is Driving Research Results Read More »
In a recent update on the 21st Century Cures Initiative, we shared how the SMA community has helped inspire this legistlation. Chairman Fred Upton has been a guest at our Hope on the Hill Congressional Dinner. He has also formed a strong relationship with Brooke and Brielle Kennedy, two sisters from his home state of
Meet the SMA Family that Helped Inspire 21st Century Cures Read More »
Cytokinetics, in partnership with Astellas, has officially announced that recruitment will begin in the second half of 2015 for a Phase 2 clinical trial testing CK-2127107, a muscle activator. This randomized, double-blind study is expected to enroll 75 patients with SMA type II, III, or IV. Patients will be dosed for four weeks, and will
Cytokinetics and Astellas Release Further Details on Upcoming Phase 2 Trial Read More »
Cure SMA has become a participating organization of The Research Acceleration and Innovation Network (TRAIN), a program of FasterCures, bringing the total number of venture philanthropies in TRAIN to 80. FasterCures established TRAIN in 2005 to connect disruptive innovators in the disease research space with the vital resources, tools and relationships to catalyze development of
Cure SMA Joins The Research Acceleration and Innovation Network Read More »
