The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
[Editor’s Note: This is part of a series of articles into the discovery and development of Evrysdi, SMA’s newly approved disease-modifying therapy and its first oral and at-home one, as well as the scope of SMA issues and treatments. Here, we talk with an Indiana man who began…
From candlelighting events to storytelling, efforts to mark Spinal Muscular Atrophy Awareness Month, observed each August, are underway to heighten awareness of the rare genetic disease and raise funds to fight it. Supporters are participating in a range of activities such as changing their social media profile photo, sharing…
The University of Birmingham has begun a study to gauge the impact of the COVID-19 pandemic on adults in the United Kingdom (UK) with neurological diseases, such as spinal muscular atrophy (SMA), that affect mobility. The series of online surveys, to be completed over 12 months, are…
The Iowa Department of Public Health has begun a pilot effort to screen newborns for spinal muscular atrophy (SMA). Babies born in Iowa are now being screened for more than 40 inherited disorders, with a goal of identifying conditions that, if caught early, can be treated or cured.
The Illinois Department of Public Health (IDPH) now includes spinal muscular atrophy (SMA) as part of its newborn screening effort. The addition of SMA — the 49th disorder in the state’s screening program — means that every baby born in Illinois will be tested for the autosomal recessive…
Adults with spinal muscular atrophy (SMA) can now receive treatment with Spinraza (nusinersen) at three new sites in England. With the addition of these centers, there are now 11 National Health Service Foundation Trust sites across the country that offer the disease-modifying therapy developed by Biogen,…
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…
The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
