We are incredibly grateful to have received a large shipment of Kinetic Sand Activity Kits to be included in the Newly Diagnosed Type II Care Packages in honor of Jayce’s second birthday! Jayce’s parents, Jenna Gomez and Corey Clark, received a care package when they contacted Cure SMA after Jayce’s diagnosis. Since receiving the care […]
Kinetic Sand Donated for Care Packages Read More »
Isis recently announced that they have tested the first patient in CHERISH, a multi-center Phase 3 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
Isis Expands Phase 3 Clinical Trials Read More »
This is an exciting time for the entire SMA community, as we celebrate another year of increasing momentum toward our goal of a treatment and cure for SMA. It’s also an exciting time for us at Cure SMA, as we’re looking forward to announcing over $1 million in new research grants in the next three
SMA Drug Pipeline Continues to Grow Read More »
The Society for Neuroscience (SfN) Meeting is the premiere neurobiology meeting, with over 30,000 scientists in attendance each year. This year’s meeting was held November 15-19 in Washington D.C. Two dozen presentations on spinal muscular atrophy were given at the meeting, several of which were by scientists funded by Cure SMA. Cure SMA-funded researchers Drs.
Cure SMA Researchers Present at Society for Neuroscience Meeting Read More »
PTC Therapeutics, Inc. announced today that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) has started a Phase 1b/2a study in adult and pediatric patients. The placebo-controlled, randomized, multiple-dose study will enroll approximately 48 patients with SMA. Because of a mutation in the survival motor neuron gene
PTC Therapeutics Announces New Phase 1a/2b Clinical Trial Read More »
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 »
