Isis recently announced the start of CHERISH, a multi-center Phase III clinical study evaluating the efficacy and safety of the investigational compound, ISIS-SMNRx, in non-ambulatory children with Spinal Muscular Atrophy (SMA). The study is a double-blind, randomized, sham-procedure controlled study in approximately 117 children with SMA at study centers in the US, Canada, Europe and
Isis Announces Second Phase III Clinical Trial Read More »
On November 15, 140 guests gathered for the Inaugural Southern California Cure SMA Gala of Hope. This fantastic evening raised over $80,000 in support of Cure SMA. Our thanks to those that attended, donated and supported this event. Special thanks to the event committee—Liz Bahrenburg, Nicole Lomonaco-Sunde, Nikki McIntosh, and Autumn Montoya—for all their hard
Today and tomorrow, Cure SMA’s Scientific Advisory Board (SAB) will be meeting in Washington D.C. Our Scientific Advisory Board is a recognized group of experts in SMA and motor neuron biology. They provide strategic and practical guidance in shaping our research programs. This meeting will focus on two main priorities: awarding up to $700,000 in
Cure SMA Scientific Advisory Board Meets in Washington D.C. Read More »
This year’s 4th Annual Hope on the Hill Congressional Dinner in Washington D.C. is less than three weeks away. At Cure SMA, we know that no single group can develop a treatment for SMA alone—it requires collaboration between academics, industry, government, and families. That is why we’re committed to tangible, meaningful efforts to strengthen and
Families, Government, and Industry Leaders Unite to Help Cure SMA Read More »
Cure SMA is our name, and it’s also our rallying cry. It’s why we keep funding research and family support. And we know it’s why our community gives so much to host events, raise awareness, lobby for policy changes, and support each other. Together, we can cure SMA. In that spirit, we’re asking everyone in
Together, We Can Cure SMA Read More »
Members of the Cure SMA Medical Advisory Council (MAC) are holding two days of meetings, November 7-8, at the Cure SMA national offices. The MAC is one of the most highly respected bodies of SMA medical and clinical experts in the US, setting the agenda for proactive, creative, and collaborative leadership on issues that improve
Medical Advisory Council Gathers for Fall 2014 Meetings Read More »
Cure SMA and BAYADA Pediatrics is proud to jointly announce that BAYADA has renewed its partnership with Cure SMA, as a national sponsor of the Walk-N-Roll program. Throughout the past year, in the true spirit of providing community service where they live and work, BAYADA Pediatrics offices came together to raise funds and awareness for
BAYADA Pediatrics Renews National Sponsorship with Cure SMA Read More »
A manuscript from the laboratory of Dr. Brian Kaspar of Nationwide Children’s Hospital was recently published in the journal Molecular Therapy. The paper, “Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA—a dose response study in mice and nonhuman primates” is the first publication resulting from a groundbreaking collaboration between Cure SMA, the
Promising Results Published on Cure SMA-Funded Gene Therapy Project Read More »
The latest issue of Compass, our research publication, is now available online. This Fall 2014 issue includes news from our 2014 SMA researcher meeting, and news from the 2014 CME conference. Also in this issue, Cure SMA Scientific Advisory Board member Arthur Burghes, PhD, provides an update from our 2014 conference special session, “Moving Beyond
Read the Fall 2014 Issue of Compass Read More »
We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. This is the last of four total updates, and the second of two in drug discovery and clinical trials. This session was moderated by Douglas Kerr, MD, PhD, Biogen Idec.
2014 SMA Researcher Meeting Summary: Clinical Trials Read More »
