Patricia Inácio, PhD,  science writer—

The HOPE-3 clinical trial testing Capricor Therapeutics’ experimental cell therapy CAP-1002 for Duchenne muscular dystrophy (DMD) can continue as planned, the company announced. That’s the result of an interim futility analysis, which evaluates whether it is mathematically possible for a trial to achieve its goal — if not, the trial…

Growth hormone (GH) or testosterone can better protect against spinal fractures than a common osteoporosis medication alone in boys with Duchenne muscular dystrophy (DMD) or severe Becker muscular dystrophy (BMD), according to a long-term study. Used to prevent or lessen osteoporosis, or weak and brittle bones, zoledronic acid (ZA) is…

The blood of boys with Duchenne muscular dystrophy (DMD) show alterations in the metabolism of fatty molecules, amino acids, and carnitine, a nutrient helping cells produce energy, relative to children without this disease, a study from China reports. Differences in the gut microbiome, the collection of microorganisms living in…

AMO-02 (tideglusib), an experimental oral therapy for certain muscular dystrophies, showed promise in boosting motor and cardiac muscle health in a mouse model of Duchenne muscular dystrophy (DMD). According to AMO Pharma, the treatment’s developer, AMO-02 also improved the animals’ metabolism and cognitive abilities. “These studies…

Blocking the activation of a channel in mitochondria, the cells’ powerhouses, may be a promising way to stop muscular dystrophy (MD) progression in a mouse model of the disease. “We have isolated the primary disease-causing component of muscular dystrophy to the mitochondrial permeability pore,” Jeffery Molkentin, PhD, co-executive director…

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 LELANTOS-1 Phase 3 clinical trial, assessing pamrevlumab in combination with corticosteroids in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, did not reach its main goal. FibroGen, the therapy’s developer, announced the top-line results from the trial, whose main objective was to assess changes in upper…

MP1032, MetrioPharm’s oral therapy candidate for Duchenne muscular dystrophy (DMD), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). A Phase 2 trial testing MP1032 in DMD patients is expected to start next year. “Currently, DMD cannot be cured, but it can be treated,”…

Dyne Therapeutics’ investigational therapy DYNE-101 has been granted an orphan drug designation by the European Medicines Agency (EMA) for myotonic dystrophy type 1 (DM1). The designation is meant to support the development of potential therapies for rare, life-threatening, or chronically debilitating diseases. The benefits include reduced fees, clinical…

AOC 1044, an investigational exon 44-skipping therapy for Duchenne muscular dystrophy (DMD), has been granted fast track status by the U.S. Food and Drug Administration (FDA). The designation is intended to accelerate the therapy’s development and expedite its approval by providing more frequent meetings with the FDA and discussions…