Author name: Morgan Tuttle

Ionis Pharmaceuticals Issues Community Statement on the Approval of Spinraza

Leave a Comment / Clinical Trials, Community Awareness, Front Page News /

Ionis Pharmaceuticals has issued the following community statement on the approval of Spinraza for SMA: Dear Members of the SMA Community, Today is a historic day for the SMA community with the approval of SPINRAZA™ (nusinersen) by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA). This is a […]

Ionis Pharmaceuticals Issues Community Statement on the Approval of Spinraza Read More »

FDA Approves Spinraza for SMA

Leave a Comment / Advocacy, Clinical Trials, Front Page News, Our Impact /

On December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA. We are thrilled to see our community’s efforts culminate in the approval of Spinraza: not only the first-ever approved treatment for this disease, but also one that addresses the underlying

FDA Approves Spinraza for SMA Read More »

Cure SMA Participates in a Special FDA Listening Session

Leave a Comment / Advocacy, Community Awareness, Front Page News, Our Impact /

Last week, Cure SMA participated in a listening session with FDA Commissioner Robert M. Califf, MD, MACC. The FDA organized the listening session in order to gain a better understanding of the current goals, priorities, and challenges of our community. Representatives from Cure SMA presented to the Commissioner and a panel of more than 25

Cure SMA Participates in a Special FDA Listening Session Read More »

Cure SMA Announces Additional Conference Support for Newly Diagnosed Families

Leave a Comment / Conference, Front Page News, Our Impact /

Thanks to the generous funding provided by the Erin Trainor Memorial Fund, Cure SMA is now offering additional support for newly diagnosed families to attend the conference. New for this year, the scholarship will now cover three hotel nights, in addition to registration fees for up to four family members, to help these families attend

Cure SMA Announces Additional Conference Support for Newly Diagnosed Families Read More »

SMA Community Conducts Outreach Visits with Members of Congress

Leave a Comment / Advocacy, Front Page News /

On November 30, members of the SMA community met with 17 different legislative offices— eight from the Senate and nine from the House of Representatives.  These Senators and Representatives serve on committees important to our community’s goals, including the House Energy & Commerce Committee; Senate Health, Education, Labor and Pensions (HELP) Committee; the House and

SMA Community Conducts Outreach Visits with Members of Congress Read More »

Jadon’s Hope Foundation Donates 15 Items to Cure SMA Equipment Pool

Leave a Comment / Support & Care /

Cure SMA recently recieved a donation of nine special tomato feeder seats and six telescopic ramps from Jadon’s Hope Foundation. The feeder seats and ramps were immediately sent out to families who were on a waiting list for either item. Jadon’s Hope Foundation was created by Tony and Kristin Burks, in honor of their son, Jadon,

Jadon’s Hope Foundation Donates 15 Items to Cure SMA Equipment Pool Read More »

The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants

Leave a Comment / Clinical Trials, Front Page News, Research /

Today, Lancet published results from the Phase 2, open-label trial of nusinersen (Spinraza) in infants. This trial initiated in 2013, and patients continue to receive the drug.  The results show that infants as young as five weeks old with the most severe form of spinal muscular atrophy (SMA) – a leading genetic cause of infant

The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants Read More »

The Miller McNeil Woodruff Foundation Makes a Generous Contribution to Cure SMA

Leave a Comment / Community Awareness, Front Page News /

The Miller McNeil Woodruff Foundation awarded $87,000 to Cure SMA, in order to further research that will lead to a treatment and cure for SMA. Their gift will also help fund continued advocacy and awareness intiatives. The generous donation is made each year by Patrick and Meredith Woodruff, in honor of thier son, Miller, who passed away from

The Miller McNeil Woodruff Foundation Makes a Generous Contribution to Cure SMA Read More »

Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on Clinical Trial Outcomes in Adults with SMA

Leave a Comment / Front Page News, Research /

Cure SMA-funded researcher, Chad Heatwole, and his team, have published a paper, “What Matters Most: A Perspective From Adult Spinal Muscular Atrophy Patients”. This project was a pilot study funded by a grant from Cure SMA, and focused on developing a reliable method for adults with SMA to report on clinical trail outcomes that are

Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on Clinical Trial Outcomes in Adults with SMA Read More »

Annual Congressional Dinner Brings SMA Community Together on the Eve of 21st Century Cure Bill Passage

Leave a Comment / Advocacy, Community Awareness, Front Page News /

On Tuesday, November 29, more than 160 people from the SMA community – including government and industry partners – gathered for the 6th Annual “Hope on the Hill” Congressional Dinner. Several Members of Congress attended the dinner, including Reps. Sean Duffy, Bill Foster, Jeb Hensarling, Bill Huizenga and Erik Paulsen. Other honored guests included U.S.

Annual Congressional Dinner Brings SMA Community Together on the Eve of 21st Century Cure Bill Passage Read More »

Scroll to Top