Evrysdi safely boosts SMN levels in previously treated patients: Trial
A year of treatment with Evrysdi (risdiplam) was safe and boosted levels of SMN — the protein that’s deficient in…
Articles by Lindsey Shapiro, PhD
Children with SMA should be monitored for cognitive function
Children with spinal muscular atrophy (SMA) type 1 who start treatment with approved therapies after their symptoms emerge…
Bicalutamide and trehalose show promise in mouse model of SBMA
Treatment with bicalutamide and trehalose extended survival and improved motor function in a mouse model of spinal and bulbar…
Targeting 2 specific proteins shows promise for SBMA in early models
A gene-silencing therapy designed to lower the levels of two proteins was found to improve muscle strength and coordination in…
Evrysdi’s motor benefits sustained for 2 years in SUNFISH study
People with spinal muscular atrophy (SMA) types 2 and 3 who received Evrysdi (ridisplam) in the SUNFISH clinical…
Ionis Sharing Royalties From Spinraza Sales for $500M Upfront
In an agreement meant to provide Ionis Pharmaceuticals with additional money to advance potential therapies for various diseases, the…
Apitegromab Clinical Trial Updates Expected This Year
The ongoing SAPPHIRE clinical trial testing apitegromab, Scholar Rock’s investigational muscle-directed therapy, in people with spinal muscular atrophy…
CANbridge Acquires Possibly Safer, More Effective SMA Gene Therapy
CANbridge Pharmaceuticals has acquired exclusive global rights to develop, manufacture, and commercialize a second-generation gene therapy for…
Biogen, Alycone Team Up for Easier ASO Treatment Device
Biogen is collaborating with Alcyone Therapeutics to develop an implantable medical device that more easily delivers antisense oligonucleotide (ASO)…
FDA OKs Phase 2 Trial of Oral Therapy NMD670 for SMA Type 3
The U.S. Food and Drug Administration (FDA) has given NMD Pharma the green light to conduct a Phase 2…