Children with Duchenne muscular dystrophy (DMD) may be more prone to develop early language abnormalities, according to a recently published study in Brain Sciences. Although DMD is typically associated with progressive muscular weakness and atrophy, there is a growing interest in understanding the role of DMD dystrophin isoforms in the central nervous system and their influence…
Registration is now open for the CureDuchenne 2023 FUTURES National Conference focused on Duchenne muscular dystrophy (DMD). The conference is “an annual event focused on bringing education, connection, and hope to the entire Duchenne community,” according to its sponsor, CureDuchenne. The conference will take place Friday, April 21 through Sunday, April 23, 2023, with a…
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Deanna Tucker, PharmD, a medical science liaison team leader at Sarepta Therapeutics, on the company’s efforts to develop a gene therapy for Duchenne muscular dystrophy. Read the full story here: Sarepta Simultaneously Pursues Exon Skipping, PPMO, and Gene Therapies for DMD
Cathelicidin-related antimicrobial peptide (CRAMP) appears to be an essential muscle degeneration mediator in mouse models and could be a potential therapeutic target for patients with Duchenne muscular dystrophy (DMD) in the future, according to a recent study. The authors aimed to assess the role of CRAMP in muscle degeneration by exposing mice to cardiotoxin barium…
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michael Kelly, PhD, chief scientific officer at CureDuchenne, on the latest developments in exon skipping, gene therapy, and other treatments for Duchenne muscular dystrophy. Read the full story here: Gene Therapy Could Emerge as a DMD Treatment in 2023
A new randomized clinical trial demonstrated that vamorolone, an experimental dissociative steroidal anti-inflammatory medication, was safe and effective in boys with Duchenne muscular dystrophy (DMD) over a 24-week period. The study results, published in JAMA Neurology, suggest that vamorolone may be a safer alternative to long-term prednisone, which is commonly prescribed in DMD. “First-in-patient, open-label,…
The siblings of patients with Duchenne muscular dystrophy (DMD) were more likely to concentrate on DMD-linked desires and aspirations associated with work, intimacy, self-image, community, family, and living with discomfort than a cohort of peers of similar ages, a recent study suggests. The results also showed that the siblings of patients with DMD were more focused…
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Paul Heaton, a Montana cattle rancher whose son has Duchenne muscular dystrophy (DMD), on his Calves2Cure DMD fundraising initiative. Read the full story here: A Unique Way Raise Money for DMD Research: Cattle Auctions
The presence of late gadolinium enhancement (LGE) on cardiac magnetic resonance imaging (MRI) before the age of 14 years in patients with Duchenne muscular dystrophy (DMD) is associated with a faster rate of cardiac decline, according to a study published in Pediatric Cardiology. Patients with an earlier onset of LGE had a more rapid reduction…
Transplantation of genetically corrected Duchenne muscular dystrophy (DMD) patient-derived induced pluripotent stem cells (DMD-iPSCs) could represent a potential therapeutic alternative for patients with DMD, according to a recently published study in the International Journal of Molecular Sciences. Currently, patients with DMD are mostly treated with corticosteroids, which delay muscle atrophy but do not prevent it. Thus,…
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