Dear SMA Community, As 2020 draws to a close, we want to provide an overview of our recent work in the community over the fourth quarter and updates related to clinical studies of Evrysdi™ (risdiplam), which was approved earlier this year for the treatment of spinal muscular atrophy (SMA) in people 2 months of age […]
Genentech End-of-Year Community Statement Read More »
Below we have provided an update from Biogen regarding the status of its DEVOTE clinical trial. For more information about SPINRAZA (nusinersen), visit https://www.curesma.org/spinraza/. ++++++++++++++++++++ Biogen’s DEVOTE study is designed to evaluate the safety, tolerability, and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved for the treatment
Biogen Provides SMA Community Update on DEVOTE Study Read More »
Brian Chiorello is a Principal Service Delivery Manager at a financial services company near where he lives in Orlando, FL. He resides there with his wife and 3-year-old daughter. In the early 1990s, Brian had plans to join the Navy, but things changed when a naval doctor suspected something was not quite right. “His suspicions
Community Spotlight: Brian Chiorello Read More »
During our past fiscal year—from July 1, 2019 to June 30, 2020—Cure SMA funded more than $9 million in new and ongoing research and care initiatives. This money will be used strategically to help accelerate research and ensure we are developing treatments and protocols for all types, ages, and stages of spinal muscular atrophy (SMA).
Cure SMA Funds Over $9 Million in New and Ongoing Research in FY20 Read More »
High school sweethearts, Steven and Amy Hoskin, and their 19-month-old son, Grayson, reside outside Sacramento, CA. Steven describes them as a goofy bunch, always playing practical jokes on each other. Grayson is quick to acclimate to his family’s cheerful demeanor and loves to join in on the silliness. Grayson has been through a lot for
Community Spotlight: The Hoskin Family Read More »
Recently, Cure SMA and colleagues at Novartis Gene Therapies published a manuscript titled, “Economic burden of spinal muscular atrophy: an analysis of claims data” in the Journal of Market Access & Health Policy. The purpose of the manuscript was to describe an analysis that was conducted measuring direct costs related to spinal muscular atrophy (SMA)
To the full spinal muscular atrophy (SMA) community, we want you to know that Cure SMA is continuing to support you with information and resources related to the COVID-19 pandemic. The following frequently asked questions (FAQs) are designed to help address key concerns expressed by members of the SMA community about COVID-19 vaccination and provide
Answering Questions about COVID-19 Vaccination and SMA Read More »
Dear SMA Community, Although this year has brought with it many challenges, the SMA community continues to demonstrate its strength and resilience. Cure SMA remains committed to bringing you the information, resources, and support you need as we continue to navigate the COVID-19 pandemic. Last week, the Advisory Committee on Immunization Practices (ACIP)—a U.S. Centers
Cure SMA Shares Perspective on COVID-19 Vaccination Read More »
