March 2015

Cure SMA Welcomes the Virginia Chapter

Cure SMA is pleased to announce the creation of a new chapter in Virginia. This chapter will also serve residents of Washington DC. Led by chairpersons Jessica White (family support), Jim Ferry (fundraising), and Kyle & Laura Derkowski (communications/awareness), the chapter is excited to begin helping families throughout their area, and fundraising to support research […]

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Human Tissue Donation Program Advances Understanding of SMA

Families’ generous donations of human tissues at the time of death are critical to an ongoing program directed to understanding how SMA damages the nervous system. One persistent mystery about SMA, of potential great importance, is why the low level of the SMN protein within all cells particularly affects motor neurons. With the tissues available

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Cure SMA Releases the Schedule for the 2015 Annual SMA Conference

Cure SMA has released the schedule for the 2015 Annual SMA Conference, which will be held in Kansas City, June 18-21. This will be the 26th year that Cure SMA has hosted this conference. The schedule includes the timeline of workshops and events, including the Meet & Greet and Family Fun Fest, the Family Friendly

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Cure SMA Announces New $140,000 Basic Research Grant to Chad Heatwole, MD, at the University of Rochester

Cure SMA has awarded a $140,000 research grant to Chad Heatwole, MD, at the University of Rochester for his project, “Development of a Clinically Relevant Outcome Measure for SMA Therapeutic Trials.” As more spinal muscular atrophy drug programs progress to clinical trials, it becomes critically important to develop ways of accurately measuring whether or not

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SMA Researcher Meeting to Host Over 100 Presentations

The Annual SMA Conference consists of two separate events that run in parallel. The first is focused on families affected by SMA, who gather for workshops, keynote sessions, networking, and fun events such as a dance party, movie night, and more. The second event is the SMA Researcher Meeting, the largest research meeting in the

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Biogen Announces NURTURE, a New Phase 2 Clinical Trial

Biogen, in collaboration with Isis Pharmaceuticals, has announced that they will soon open recruiting for NURTURE. This is a multi-center, Phase 2 clinical study evaluating the efficacy of the investigational drug, ISIS-SMNRx (ISIS 396443), in pre-symptomatic newborns that have a genetic diagnosis of Spinal Muscular Atrophy (SMA). The purpose of the NURTURE study is to

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