Federal Committee to Vote on Spinal Muscular Atrophy Newborn Screening on Thursday

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On Thursday, February 8, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), a federal committee that oversees newborn screening, will vote on whether to recommend newborn screening for spinal muscular atrophy. This decision is an important step toward our goal of having every baby born in the United States screened for SMA.

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Preliminary Data from FIREFISH Trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy

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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

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AveXis Releases Community Statement on Expanded Clinical Trials

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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

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Cure SMA to Host Webinar on Treatment Access and Clinical Trials on February 15

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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

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SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report

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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

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2018 Updated SMA Drug Pipeline Released

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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

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AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy

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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

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Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials

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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

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Cure SMA Launches Advocacy Action Network

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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

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Minnesota Becomes Second State to Adopt Newborn Screening for SMA

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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

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