Biogen has provided the following update on resources they have made available for the SMA community: Biogen recently announced that the U.S. Food and Drug Administration has approved SPINRAZA™ (nusinersen) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. At Biogen, we understand that there are many challenges that families living […]
Biogen Resources for the SMA Community Read More »
Please join Cure SMA, MDA and representatives from Biogen to learn more about SPINRAZA™ (nusinersen). As you know, SPINRAZA recently received FDA approval in the U.S. for the treatment of Spinal Muscular Atrophy (SMA) in pediatric and adult patients. In order to receive an update on SPINRAZA, please join this webcast, which is intended only
Join Us for an Upcoming Webcast about SPINRAZA TM (nusinersen) Read More »
Biogen provided the following statement on the approval of Spinraza for SMA: Dear Members of the SMA Community, Today, the SMA community achieved a goal that would not have been possible without the support and dedication of the families, physicians and researchers who have passionately pursued a treatment for Spinal Muscular Atrophy (SMA). We are
Biogen Provides Community Statement and Q&A on the Approval of Spinraza for SMA Read More »
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 »
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 »
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 »
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 »
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 »
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 »
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
