Some advice on reading facial expressions and body language

I recently talked with a friend about body language and facial expressions. She’d attended a public hearing where she ended up sitting just behind the main speaker. As the hearing progressed, she became certain she’d be seen on Zoom. That made her extremely conscious of her facial expressions and body…

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 (DMD), 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 (DMD). Priority review, which shortens the consideration period to six months from 10, is given to therapies with…

The toddler was crying, hungry for a snack, standing just behind me as I sliced pieces of watermelon. But I was smiling. The big kids were in the pool, and I knew they’d be delighted to have watermelon on the deck. I cracked open the sliding glass doors and yelled,…

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…

I was blessed to be able to provide music for the annual gathering of the United Church of Christ’s Penn West Conference earlier this month. Everything went well. My wife, Wendy, had plenty of help moving my equipment into the venue. The technical staff from PennWest California did…

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…

While the boys are away, it’s time to play! Well, not exactly. My three sons with Duchenne muscular dystrophy (DMD) — Max, 17, Rowen, 14, and Charlie, 12 — are away this week at Muscular Dystrophy Association Summer Camp. But instead of rest, my husband and I are…

Catalyst Pharmaceuticals has entered a licensing agreement for the North American rights to vamorolone, a dissociative corticosteroid under review in the U.S. and Europe for Duchenne muscular dystrophy (DMD). Under the deal, Catalyst will give vamorolone’s developer Santhera Pharmaceuticals an upfront cash payment of $75 million,…

The U.S. Food and Drug Administration (FDA) granted accelerated approval to the gene therapy SRP-9001 (delandistrogene moxeparvovec) for children ages 4 to 5 with Duchenne muscular dystrophy (DMD) who can walk and have a confirmed mutation in the DMD gene. The treatment is now named Elevidys (delandistrogene moxeparvovec-rokl). “The approval…