AveXis, a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an overview of the expanded clinical development program for the company’s initial gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy (SMA). In addition to the ongoing pivotal trial in SMA Type 1 […]
Scholar Rock, a biotechnology company based in Boston, has announced that they will be investing nearly $50 million to bring SRK-015, a muscle drug for possible treatment of SMA, into human clinical trials. SRK-015 is Scholar Rock’s lead clinical candidate. They expect to start clinical development in the first half of 2018, although timelines for
Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials Read More »
Working together, we’ve achieved tremendous success in advocating for ourselves, our families, and our whole community, particularly in the past few years. Today, we’re building on this momentum with the launch of our Advocacy Action Network. The Advocacy Action Network will allow our advocates to share their voices on issues that impact our community, including research
Cure SMA Launches Advocacy Action Network Read More »
On December 27, the Minnesota Commissioner of Health accepted the recommendation of the state’s Advisory Committee on Heritable and Congenital Disorders to add SMA to Minnesota’s newborn screening panel. With this decision, Minnesota becomes the second state, after Missouri, to adopt permanent newborn screening for SMA. Cure SMA will also be working with an implementation
Minnesota Becomes Second State to Adopt Newborn Screening for SMA Read More »
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 »
