The complexities of newborn screening require a sophisticated, multifaceted approach. With this in mind, Cure SMA has been working on newborn screening through several different avenues, bringing our full resources to ensure that babies born with SMA have the opportunity to receive early treatment for maximum effectiveness. Advocacy We are working with family advocates, clinicians, […]
Cure SMA’s Approach to Newborn Screening Read More »
The first FDA approval of a therapy for SMA has created an opportunity for our community to move forward on another of our long-term priorities: newborn screening. SMA is the leading genetic cause of death for infants, but we have an historic opportunity to change that through newborn screening and early treatment. Currently, the average
SMA Newborn Screening Advancements Read More »
New York State will screen every child for SMA beginning October 1, according to the New York State Department of Health. New York joins Missouri, Utah, Minnesota, Indiana and Illinois as states that have adopted permanent screening for SMA. Since 2016, New York has been screening for SMA on a pilot basis. The data provided
New York to Implement Newborn Screening for SMA October 1st Read More »
Clinical trials for spinal muscular atrophy (SMA) help researchers answer important questions about the disease and investigational drugs, providing information that may help the development of future medical treatments. Although there is now an approved treatment, clinical trial participation may be a consideration for many families and individuals affected by SMA. Funding for clinical trials
What to Expect in a Clinical Trial Read More »
Cure SMA is excited to announce that 12 new Panthera pediatric lightweight wheelchairs have been purchased thanks to generous funding from Sweet Baby Zane. These specific chairs are normally a large out-of-pocket expense for many families, as they cost over $6,000 each and are not usually covered by insurance because they are not manufactured in
Sweet Baby Zane Funds New Equipment Pool Item Read More »
Cure SMA would like to thank the Cash Scanlon-Philips Foundation for their generous donation to fund a years’ worth of type II newly diagnosed care packages. These care packages are sent to every newly diagnosed SMA family, within the US after connecting with Cure SMA. These care packages include toys and helpful items for children with
Cure SMA Receives Generous Donation from the Cash Scanlon-Philips Foundation Read More »
In a recent paper titled, “Patient Reported Impact of Symptoms in Spinal Muscular Atrophy (PRISM-SMA)” Dr. Chad Heatwole and others describe a cross-sectional study of 359 adults with SMA using the International SMA Patient Registry. The goal of the study was to determine the frequency and relative importance of symptoms experienced by adults with spinal
Quinn Anton Jensen was diagnosed with spinal muscular atrophy (SMA) type II on March 2007 in Iowa City. Quinn’s mom, Nancy, recalls the genetic testing process took over a month. It was a stressful and frightening time and the Anton Jensens wish the diagnosis process was faster. Nancy recalls feeling devasted as there was still
Community Spotlight: The Anton Jensen Family Read More »
Mary was 4 months old when her parents, Ryan and Kate Nelson, suspected something wasn’t quite right with their daughter; she wasn’t bearing weight on her legs nor was she lifting her head like their friends’ babies. Mary’s pediatrician referred them to a neurologist who presumed Mary had a stroke in utero, so he ordered
Community Spotlight: The Nelson Family Read More »
At 7 months old, Sarah Zmaczynski’s parents, Jill and John, noticed their daughter couldn’t bear weight on her legs. Sarah’s pediatrician responded with the “kids develop at different rates” speech. As John has a family history of late walkers, the Zmaczynski’s thought maybe their worries were unfounded. Although one in 50 people carry the genetic
Community Spotlight: The Zmaczynski Family Read More »
