The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AOC 1044, an experimental exon 44-skipping therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Avidity Biosciences. Orphan drug designation is given to therapies with the potential to treat rare disorders, those that affect fewer…
A new project hopes to improve data collection related to Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), with the aim of accelerating research for both diseases. The effort is a collaboration between the Duchenne Data Foundation (DDF) and the Duchenne Regulatory Science Consortium (D-RSC), a…
A 5-year-old boy has received an  infusion of Elevidys, the recently approved gene therapy for Duchenne muscular dystrophy (DMD), at Nationwide Children’s Hospital, where the treatment was first investigated. Gideon Griffiths of Bellefontaine, Ohio, who was diagnosed with DMD at birth, is among the first group of patients nationwide…
The first person with limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9) has been dosed with the gene therapy AB-1003 in a clinical trial, according to an announcement from the therapy’s developer Asklepios BioPharmaceutical (AskBio). “The first limb-girdle muscular dystrophy 2I/R9 patient dosed in the LION-CS101 clinical trial is…
The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease status to SAT-3153, Satellos Bioscience’s first-in-class therapy candidate to rebuild muscle tissue in Duchenne muscular dystrophy (DMD). “Receiving orphan drug designation and rare pediatric disease designations are important milestones in advancing our…
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy status to NS-089/NCNP-02, NS Pharma’s candidate for Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping, the company announced. The designation is intended to speed up the development and review of therapies for serious or life-threatening…
Various factors influenced the results of the creatine kinase-MM (CK-MM) blood test used to screen infants for Duchenne muscular dystrophy (DMD), according to a large study. Factors that affected CK-MM levels included age at blood sample collection, gestational age, birth weight, sex, ethnicity, and seasonal temperature, data showed. Newborn…
Mental health medications, including the stimulant methylphenidate, may help ease neurobehavioral problems in boys and young men with Duchenne muscular dystrophy (DMD), according to a small study by researchers in Belgium and the Netherlands. However, some adverse reactions…
Ahead of pending regulatory decisions for vamorolone — a treatment for Duchenne muscular dystrophy (DMD) now under review in the U.S. and Europe — Catalyst Pharmaceuticals has fully acquired the commercial rights to the therapy in North America. The finalized deal comes about a month after Catalyst…
Arrowhead Therapeutics has asked for permission to launch a clinical trial of ARO-DUX4, an experimental RNA-based medicine for facioscapulohumeral muscular dystrophy (FSHD) type 1, in New Zealand. The company’s application, filed with the New Zealand Medicines and Medical Devices Safety Authority, will be reviewed by the Standing…
Get regular updates to your inbox.