October 2017

Fall 2017 Issue of Compass Now Available

The fall 2017 issue of Compass is now available online. This issue covers Phase II of the SMA Industry Collaboration, as well as highlights and results from the Patient Focused Drug Development Meeting with the FDA. Cure SMA Launches Phase II of the SMA Industry Collaboration The SMA Industry Collaboration is a multi-faceted partnership that […]

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Cure SMA Launches Benefit-Risk Survey to Address Important Issues in Treatment Access and Future Drug Approvals in SMA

For the past several years, we have been working to bring your voices, experiences, challenges and hopes to inform regulatory authorities as key decisions are made on future therapies for SMA. Most recently, on April 18, 2017, the SMA community gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA to do just that.

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Cure SMA Announces Over $5 Million in New Research and Care Funding

Cure SMA is pleased to announce $5 million in new research funding over the next 12 months. This funding will be used strategically to help accelerate research and ensure we are developing treatments for all types, ages and stages of SMA. This funding will also be used to increase patient access to treatments and improved

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Registration is Now Open for the 2018 Annual SMA Conference!

Registration is now open for the 2018 Annual SMA Conference! This year’s conference will be held at the Hilton Anatole Hotel in Dallas, TX, from Thursday, June 14 through Sunday, June 17, 2018. We are excited to bring the conference to a central location and hope you can join us! Located in the Market Center

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Cure SMA Meets in Utah with State and Federal Elected Officials to Support Newborn Screening

On Tuesday, Cure SMA staff members and family advocates, and clinicians from the University of Utah, met with Senator Orrin Hatch (R-UT) to discuss newborn screening for spinal muscular atrophy. Senator Hatch was an original sponsor of the Newborn Screening Saves Lives Act of 2007, which established federal programs to support states in the implementation

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Genentech/Roche Releases Clinical Trial Update for SUNFISH (RG7916)

Genentech/Roche has provided the below community statement with clinical trial updates for SUNFISH (Type 2/3). The first patient is now enrolled in SUNFISH, as the trial advances to part two, which is designed to be a pivotal marketing registration trial.  Dear SMA Community, We are pleased to share an update on the SUNFISH study evaluating

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