Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial called EMERGENE that will test its gene…
Articles by Patricia Inácio, PhD
Agamree (vamorolone) has been approved in the U.K. for treating Duchenne muscular dystrophy (DMD) in patients 4 and…
Disease progression with Becker muscular dystrophy (BMD) varies considerably, and depends on a patient’s age — with progression largely…
The HOPE-3 clinical trial testing Capricor Therapeutics’ experimental cell therapy CAP-1002 for Duchenne muscular dystrophy (DMD) can continue as planned,…
Growth hormone (GH) or testosterone can better protect against spinal fractures than a common osteoporosis medication alone in boys with…
The blood of boys with Duchenne muscular dystrophy (DMD) show alterations in the metabolism of fatty molecules, amino acids,…
AMO-02 (tideglusib), an experimental oral therapy for certain muscular dystrophies, showed promise in boosting motor and cardiac muscle…
Blocking the activation of a channel in mitochondria, the cells’ powerhouses, may be a promising way to stop muscular…
The U.S. Food and Drug Administration has granted rare pediatric disease status to NS Pharma‘s NS-089/NCNP-02, an investigational exon…
The LELANTOS-1 Phase 3 clinical trial, assessing pamrevlumab in combination with corticosteroids in people with Duchenne muscular dystrophy (DMD) who are no longer…