The winter 2018 issue of Compass is now available online. This issue covers Cure SMA’s community survey, and reviews how data is used to improve research, care and coverage in the SMA community. Cure SMA Community Update Survey Cure SMA has been collecting data through our database and various projects over the past several years. […]
Winter 2018 Compass Now Available Online Read More »
The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) today recommended that newborn screening for spinal muscular atrophy be implemented nationwide. This decision is an important step toward our goal of having every baby born in the United States screened for SMA. The recommendation now goes to Health and Human Services Secretary Alex
The Utah Department of Public Health has announced that, effective immediately, all newborns born in the state will be screened for spinal muscular atrophy. This announcement makes Utah the first state to implement permanent SMA screening. Late last year, the Utah Newborn Screening Advisory Committee recommended that SMA be added to the state screening panel.
Utah Becomes First State to Implement Permanent Screening for SMA Read More »
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.
Federal Committee to Vote on Spinal Muscular Atrophy Newborn Screening on Thursday Read More »
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
