PTC Therapeutics, Inc. recently announced the presentation of early interim data from Part 1, the dose-finding portion of the FIREFISH study. FIREFISH is a two-part seamless, open-label, multi-center study to investigate the safety and efficacy of RG7916 in infants and babies with Type 1 SMA. RG7916 has been safe and well tolerated at all doses […]
AveXis has provided the following community statement on AVXS-101. Dear SMA Community, At AveXis, the gene replacement therapy company developing a new approach to treat SMA known as AVXS-101, we recently announced our plans to start three new clinical trials (STR1VE EU, SPRINT, REACH) in addition to our ongoing clinical trials (STR1VE, STRONG). The goal
AveXis Releases Community Statement on Expanded Clinical Trials Read More »
On Thursday, February 15, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST), Cure SMA will hold a webinar updating the community on treatment access and clinical trials. Among the topics covered will be: The current status of Spinraza access, including dosing, sites, and insurance Updates on ongoing clinical trials Updates on upcoming clinical trials News from
Cure SMA to Host Webinar on Treatment Access and Clinical Trials on February 15 Read More »
Cure SMA and our partners in the SMA Industry Collaboration are pleased to announce the release of the Spinal Muscular Atrophy Voice of the Patient (VoP) Report. This report is a thorough written account of the Patient-Focused Drug Development (PFDD) Meeting with the FDA, in the spring of 2017. Patient Focused Drug Development Meeting with
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 16 active programs, including one approved therapy. 14 pharmaceutical partners. 6 programs in clinical trials. An ever-increasing breadth of potential treatment approaches to SMA. Effective Treatments for All Ages, Stages and Types of SMA In December 2016, our community celebrated the approval
2018 Updated SMA Drug Pipeline Released Read More »
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 »
