Opportunities and challenges for antisense oligonucleotide therapies
Kuijper EC, Bergsma AJ, Pim Pijnappel WWM, Aartsma-Rus A. Opportunities and challenges for antisense oligonucleotide therapies. J Inherit Metab Dis…
Noninvasive Prenatal Diagnosis for Duchenne Muscular Dystrophy Based on the Direct Haplotype Phasing
Chen M, Chen C, Huang X, et al. Noninvasive prenatal diagnosis for Duchenne muscular dystrophy based on the direct haplotype…
Blood-derived biomarkers correlate with clinical progression in Duchenne muscular dystrophy
Strandberg K, Ayoglu B, Roos A, et al. Blood-derived biomarkers correlate with clinical progression in Duchenne muscular dystrophy. Journal of…
Rehabilitation Following Fracture in Dystrophinopathy, A Case Series
Glanzman AM, Jones J, Thompson CZ, et al. Rehabilitation Following Fracture in Dystrophinopathy, A Case Series. Journal of neuromuscular diseases…
Combining genetics, neuropsychology and neuroimaging to improve understanding of brain involvement in Duchenne muscular dystrophy - a narrative review
Doorenweerd N. Combining genetics, neuropsychology and neuroimaging to improve understanding of brain involvement in Duchenne muscular dystrophy - a narrative…
Comparison of Survival Analysis between Surgical and Non-surgical Treatments in Duchenne Muscular Dystrophy Scoliosis
Yang JH, Kim KS, Lee GH, Kim HS. Comparison of Survival Analysis between Surgical and Non-surgical Treatments in Duchenne Muscular…
Viltolarsen: First Approval
Dhillon S. Viltolarsen: First Approval. Drugs 2020;80:1027-1031 Abstract Viltolarsen (Viltepso((R)) in Japan) is a phosphorodiamidate morpholino antisense oligonucleotide being…
Natural history of serum enzyme levels in Duchenne muscular dystrophy and implications for clinical practice
Rodriguez-Cruz M, Almeida-Becerril T, Atilano-Miguel S, Cardenas-Conejo A, Bernabe-Garcia M. Natural history of serum enzyme levels in Duchenne muscular dystrophy…
Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy
Wagner KR, Abdel-Hamid HZ, Mah JK, et al. Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular…
Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis
Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifiro G. Global epidemiology of Duchenne muscular dystrophy: an updated…
