Cure SMA is pleased to announced that, thanks to a generous grant from The Dhont Family Foundation, we are offering scholarships to the 2017 Annual SMA Conference for all adults with SMA. The scholarship covers registration for one adult, age 18+, and one aide. Each year, we look forward to reuniting as a community at […]
Adult Scholarships Offered for the 2017 Annual SMA Conference Read More »
Registration is now open for industry and academic researchers to attend our 2017 SMA Researcher Meeting, which will be held June 29-July 1 at Disney World in Orlando, FL. The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. The 2016 SMA Researcher Meeting set a record, with over
Registration is Now Open for the 2017 SMA Researcher Meeting Read More »
Earlier this year, Cure SMA initiated an insurance coverage and payment policy project, in partnership with District Policy Group. The goal of this project is to help facilitate a favorable public and commercial coverage and payment environment. Launched in anticipation of FDA approval of new SMA treatments, the project will cover the full range of
Cure SMA Launches Insurance Coverage and Payment Policy Project Read More »
The 6th Annual South Florida Chapter Gala of Hope will be held on November 17, 2016, at Ferrari Maserati of Fort Lauderdale, Florida. The event has raised $700,000 over the last 5 years, and organizers Fiorenna Fuentes Israel and Jennifer Miller Smith have set a goal of raising $200,000 for this year’s event. The evening
6th Annual South Florida Gala of Hope Inspires Familes and Supporters Read More »
Cure SMA will be participating in a one-day workshop with The European Medicines Agency (EMA—the European equivalent of the FDA), SMA Europe and TREAT-NMD to discuss, support and advance the development of therapies for the treatment of SMA. The workshop will convene key stakeholders—patients, doctors, industry representatives, researchers and regulators—to take stock of the latest
Cure SMA Joins SMA Europe and TREAT-NMD for Meeting with EMA Regulators Read More »
On Tuesday, December 6, Cure SMA will be hosting a one-hour webinar on the New Drug Application (NDA) process. The webinar will be held at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST). Jill Jarecki, chief scientific officer, and Kenneth Hobby, president, will be joined by a two clinical and regulatory experts to discuss the NDA process
Cure SMA to Host Webinar on the New Drug Application Process Read More »
Biogen and Ionis Pharmaceuticals have provided the following community statement regarding the interim analysis of the CHERISH trial: Dear Members of the SMA Community, Today we take another important step to advance the first potential treatment option for Spinal Muscular Atrophy (SMA). We are happy to continue to provide exciting news about the SPINRAZA (nusinersen)
Biogen and Ionis Community Statement on Interim Analysis of CHERISH (Type 2) Trial Read More »
Biogen and Ionis Pharmaceuticals announced that SPINRAZA (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA. The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function
AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the planned pivotal study of AVXS-101 in spinal muscular atrophy (SMA) Type 1 will reflect a single-arm design, using natural history of the disease as a comparator, and enroll approximately 20 patients. This
AveXis Announces Single-Arm Design for Pivotal Study of AVXS-101 Read More »
Registration is now open for the 2017 Annual SMA Conference! The conference will be held at Disney’s Contemporary Resort at Disney World in Orlando, FL, from Thursday, June 29, through Sunday, July 2, 2017. We are excited to experience Disney together for a second straight year. Disney characters will be at many of our events,
Family Registration for the 2017 Annual SMA Conference is Now Open Read More »
