Higher-dose Spinraza Trial Now Enrolling Part B After No Safety Issues Found
After finding no safety concerns in Part A of the DEVOTE trial, which is testing higher doses of Spinraza (nusinersen)…
Articles by Forest Ray PhD
Lung Health Declines in Step With Motor Loss in Types 2 and 3, Study Finds
Declines in lung function are steeper and quicker in children with spinal muscular atrophy (SMA) type 2 than non-ambulatory…
Evrysdi Approved in Brazil for SMA, But No Indications Specified
Evrysdi (risdiplam) has been approved for the treatment of spinal muscular atrophy (SMA) in Brazil by the country’s…
Same But Different Photo Contest Celebrates People With Rare Diseases
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography…
FDA Approves Catalent Facility to Aid in Zolgensma’s Production
The U.S. Food and Drug Administration (FDA) has given Catalent Biologics approval to aid in manufacturing products for…
Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the…
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure…
SMN Protein Levels in Blood May Mark SMA Severity, Help Guide Treatment
Levels of the survival motor neuron (SMN) protein in the blood of people with spinal muscular atrophy (SMA)…
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during…
Coalition Will Address Racial Disparities in Rare Disease Communities
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the…