Author Archives: Marta Figueiredo PhD

DMD Adults Added to SSA Compassionate Allowances Program

The U.S. Social Security Administration (SSA) has added Duchenne muscular dystrophy (DMD) to its Compassionate Allowances (CAL) program list, accelerating the disabilities application process for adults with this genetic disorder. The program is designed to identify severe medical diseases and conditions that automatically meet the SSA’s standards for…

DMD Trial Testing Oral ASP0367 Recruiting Boys 8–16 in US

A Phase 1b clinical trial evaluating Astellas Pharma’s ASP0367 is recruiting boys with Duchenne muscular dystrophy (DMD), ages 8–16, at six U.S. sites. The ongoing trial (NCT04184882) is evaluating the safety and tolerability of the investigational oral therapy, which is designed to improve muscle function in patients with DMD,…

CRISPR Gene Editing Therapy Is Promising in New Mixed Model

A CRISPR-Cas9-based gene editing therapy promoted the production of a smaller but functional version of the dystrophin protein in a new mixed mouse model that uses muscle cells derived from Duchenne muscular dystrophy (DMD) patients. The therapy is designed to delete a DMD gene region that is commonly mutated in people…