Delpacibart zotadirsen (del-zota), an investigational exon-skipping therapy from Avidity Biosciences, is demonstrating the potential to reverse disease progression in…
Articles by Lindsey Shapiro, PhD
Researchers have developed a detailed molecular model for the structure of type VI collagen and identified where genetic mutations linked…
Ifetroban, Cumberland Pharmaceuticals’ investigational oral therapy, improved heart function and reduced cardiac damage biomarkers in people with Duchenne…
The one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) was associated with stabilizations in motor function for boys with Duchenne…
Capricor Therapeutics’ investigational therapy deramiocel, formerly CAP-1002, which is being considered for approval in the U.S., continued to…
The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to…
The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1…
A gene-editing therapy designed to correct a defect in the DYSF gene — one that’s associated with a form of…
Arrakis Therapeutics’ investigational RNA-targeted small molecule (rSM) therapies were found to work as intended in preclinical studies and eased…
A single low dose of the investigational gene therapy BB-301 helped with swallowing for the first two people with…