Yesterday, the House of Representatives passed the American Health Care Act (AHCA). The House passed legislation included an amendment that increases funding to mitigate a potential rise in health care premiums for those with pre-existing conditions; however, concerns remain about the impact this bill could have on SMA patients’ access to care (see previous update […]
Update on the Passage of the AHCA Read More »
After a failed attempt in March, Members of Congress are trying once again to assemble legislation to repeal key parts of the Patient Protection and Affordable Care Act (ACA). The latest attempt, called the American Health Care Act (AHCA), could impact SMA patients’ access to health care services by allowing states to opt out of
Cure SMA Grassroots Alert – AHCA Read More »
Cure SMA has awarded a $140,000 research grant to Christine DiDonato, PhD, Ann & Robert H. Lurie Children’s Hospital of Chicago, for her project, “Skeletal muscle activators as potential modulators of muscular weakness in SMA.” In SMA, the ability of the muscle to contract is impacted. This proposal focuses on skeletal muscle proteins and their
Biogen will present Phase 3 end of study SPINRAZA® (nusinersen) data from CHERISH, which demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset (most likely to develop Type 2 or Type 3) spinal muscular atrophy (SMA) compared to untreated children. The overall findings continue to support the robust
Biogen Releases New Data from Cherish and Nurture Trials Read More »
On April 18, 2017, the SMA community—families, clinicians, researchers, industry and regulators—gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA. As part of the reauthorization of the Prescription Drug User Fee Act (PDUFA), the FDA is required to gather community feedback more systematically, through events such as this PFDD meeting, and incorporate that
On Thursday, May 4, at 1:00pm EST (10:00am PST/11:00am MST/12:00pm CST), Cure SMA will hold a webinar updating the community on the status of Spinraza access. Among the topics covered will be: The current status of dosing in states across the US Administration sites, including information on the recent launch of our site list Commercial
Cure SMA to Host Webinar on Spinraza Access, as List of Administration Sites is Released Read More »
The European Medicines Agency (EMA) has recommended granting a marketing authorization in the European Union (EU) for Spinraza. The recommendation indicates that Spinraza should be covered for all types of SMA. This recommendation is the first step in a three-step process to bring Spinraza to European patients. Next, the EMA’s recommendation will go to the
Cure SMA has awarded a $75,000 research grant to Jean Giacomotto, PhD, at the University of Queensland, for his project, “Zebrafish models of Spinal Muscular Atrophy optimized for chemical genetics and drug discovery. From proof-of-principle to new insights and treatments”. In SMA, the lack of additional “druggable” targets, beyond SMN, creates a gap in the
Cure SMA, along with collaborators from Biogen and the SMA Foundation, have published a paper in the journal BMC Neurology. In recent years, we’ve completed 16 focus group sessions and 37 interviews in the US with 96 participants including: 21 with individuals with SMA; 64 parents of individuals affected by SMA; and 11 clinicians who
Cure SMA Publishes Second Journal Article on the Patient Voice Read More »
Cure SMA has awarded a $30,000 research grant to Remy Bordonne, PhD, at CNRS-Centre National de Recherche Scientifique, for his project, “Identification of the protective mechanism of a SMN modifier gene using S. pombe as a model organism”. In SMA, actin, which is a protein that helps muscles to contract, doesn’t work properly. The goal
