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 (Â鶹Éç) 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…
News
Various factors influenced the results of the creatine kinase-MM (CK-MM) blood test used to screen infants for Duchenne muscular dystrophy (Â鶹Éç), 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 (Â鶹Éç), 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 (Â鶹Éç) 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…
Synthetic biology company bit.bio has launched two new human muscle cell models to advance research into new treatment strategies for Duchenne muscular dystrophy (Â鶹Éç). The two models, dubbed ioSkeletal Myocytes Â鶹Éç Exon 44 Deletion, and ioSkeletal Myocytes Â鶹Éç Exon 52 Deletion, are the eighth and ninth products that…
The U.S. Food and Drug Administration has granted rare pediatric disease status to NS Pharma‘s NS-089/NCNP-02, an investigational exon 44-skipping therapy for Duchenne muscular dystrophy (Â鶹Éç), the company announced. The designation is intended to spur treatments for any rare disease (those affecting fewer than 200,000 people in…
The U.S. Food and Drug Administration (FDA) has accepted and given priority review to Italfarmaco Group’s application requesting that oral givinostat be approved to treat Duchenne muscular dystrophy (Â鶹Éç). Priority review, which shortens the consideration period to six months from 10, is given to therapies with…
After a year of treatment with the experimental oral therapy EDG-5506 in a Phase 1 study, most men with Becker muscular dystrophy (BMD) show stable or improved motor function — a marked contrast to the disease’s natural course, where motor function worsens over time. One-year findings in the…
The U.S. Food and Drug Administration (FDA) has cleared Benitec Biopharma to start a Phase 1b/2a clinical trial of the investigational gene therapy BB-301 for swallowing difficulties associated with oculopharyngeal muscular dystrophy (OPMD). Dosing is expected to start later this year, following the rollover of participants in…
Recent Posts
- I’m taking steps that will lead me away from caregiver burnout
- I almost lost my garden to Becker MD. One tool brought it back.
- New milestones hit in MD trial testing treatment to turn off faulty gene
- A Purple Parade profile amplifies my disability advocacy
- Annual campaign raises $641K to send youths to Â鶹Éç Summer Camp