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
Cure SMA would like to thank the Fighting for Kaiden Foundation for their generous donations to our equipment pool and research programs. The foundation’s generosity will provide support and hope to all those effected by SMA. The foundation recently donated two large size 2 EASyS strollers, one small size 1 EASyS stroller, and two large
Cure SMA Receives Generous Donations from Fighting for Kaiden Foundation Read More »
Abstract submission is now closed for the 2017 SMA Researcher Meeting. We are excited to announce that we received 125 submissions, a record number. Our submissions come from researchers in 14 countries, 10 companies, and close to 50 institutions. We expect over 300 researchers and clinicians from almost 100 organizations will attend the SMA Researcher
Record Number of Abstracts Submitted for the 2017 SMA Researcher Meeting Read More »
Cytokinetics, Inc. today announced that the second cohort of the Phase 2 clinical trial of CK-2127107 in patients with spinal muscular atrophy (SMA), is open to enrollment. This clinical trial is designed to assess the effect of CK-2127107, a next-generation fast skeletal troponin activator (FSTA), on multiple measures of muscle function in both ambulatory and
The Cure SMA Coverage and Payment Policy Project has two distinct but complementary tracks: Outreach on behalf of our community. We’re working with public and private insurers, regulators, and other stakeholders, advocating for prompt, comprehensive coverage for SMA treatments and care. Outreach to our community. We’re working to provide the necessary tools and information so
Cure SMA Coverage and Payment Policy Project Continues to Move Forward Read More »
