Cure SMA is pleased to announce the first grants from $450,000 in funding to help increase capacity at SMA treatment sites across the US. In order to increase the number of affected individuals that sites can follow, treat and evaluate, Cure SMA will award $50,000 grants to nine sites, for a total of $450,000 in
Cure SMA Announces Funding for Care Centers Across the US Read More »
Biogen and Ionis Pharmaceuticals announced that they have entered into a new collaboration agreement to identify new antisense oligonucleotide drug candidates for the treatment of spinal muscular atrophy (SMA). Biogen will have the option to license therapies arising out of this collaboration and will be responsible for their development and commercialization. “We are pleased to
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 Assists Hundreds of Families in 2017 Read More »
The Miller McNeil Woodruff Foundation recently awarded $87,000 to Cure SMA, in order to further research that will lead to treatments for all ages, stages and types of SMA. Their gift will also help fund continued advocacy and awareness initiatives. The generous donation is made each year by Patrick and Meredith Woodruff, in honor of
Miller McNeil Woodruff Foundation Awards Cure SMA Generous Gift Read More »
On Wednesday, Congress passed the most sweeping tax bill in a generation. Cure SMA has been monitoring and advocating on your behalf as this legislation went through the Congress. We are happy to share that: Tax credits for rare disease drugs are saved. Cure SMA partnered with other rare disease groups to save a tax
Cure SMA Statement on Tax Reform Bill and the Repeal of the Individual Mandate Read More »
The Fall 2017 issue of Directions is now available. Please visit our Support & Care Publications page to download this issue or past issues of Directions. Our biannual newsletter builds our community by sharing stories and experiences. Families from all over the world connect with each other through Cure SMA to organize, share stories, photos,
Fall 2017 Directions Now Available for Download Read More »
Over our past fiscal year—from July 1, 2016, to June 30, 2017—Cure SMA has funded 28 projects with over $2.5 million in new research funding. This funding will be used strategically to help accelerate research, and ensure we are developing treatments for all types, ages, and stages of SMA. The areas funded include: Basic research,
2017 Research Year-in-Review Read More »
SMA advocates across the country are working tirelessly with Cure SMA to educate state decision makers on newborn screening. In Maryland and Minnesota, the states’ Newborn Screening Advisory Committees voted to approve a recommendation to add SMA to the state panel. We now await final approval by both states’ Commissioners of Health. Illinois, Ohio and
Cure SMA Advocates Continue to Advance Newborn Screening Efforts Read More »
AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that, based on review of data submitted, the company may initiate its planned Phase 1 clinical trial of AVXS-101 for patients with spinal
Cure SMA is pleased to offer scholarships to the 2018 Annual SMA Conference for all adults with SMA, thanks to a generous grant from The Dhont Family Foundation. The scholarship covers registration for one adult, age 18+, and one aide. Each year, we look forward to reuniting as a community at the Annual SMA Conference.
Adult Scholarships Offered for the 2018 Annual SMA Conference Read More »
