Japan Approves Evrysdi as First At-home, Oral SMA Treatment
Evrysdi (risdiplam) has been approved in Japan as an oral, at-home treatment of spinal muscular atrophy (SMA). The flavored liquid therapy…
Articles by Patricia Inacio, PhD
Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
Asuragen’s Lab Test Can Speed Up Diagnosis of SMA Carriers, Patients
Asuragen’s lab test for spinal muscular atrophy (SMA) is as robust and accurate at identifying carriers of the…
Muscle-directed Therapy Apitegromab Put on FDA’s Fast Track
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was granted a fast track designation by…
EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
Australia Offers Subsidized Coverage of Spinraza for Pre-symptomatic Children With SMA
Australia is expanding access to Spinraza (nusinersen) to children and infants with severe spinal muscular atrophy (SMA) who have not yet developed…
Plans Advance for Biomarkers Panel to Assess Drug-induced Skeletal Muscle Injury
A new panel of four safety biomarkers to assess acute drug-induced skeletal muscle injury in Phase 1 clinical trials has…
Asuragen’s Diagnostic Test for SMA Approved for Use in Europe
Asuragen’s diagnostic test for the analysis of genes associated with spinal muscular atrophy (SMA) — called AmplideX…
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
SRK-015 Data Continue to Support Potential for SMA, Scholar Rock Says
Preclinical and clinical data on SRK-015 continue to support the therapy’s potential to treat spinal muscular atrophy (SMA),…