Duchenne muscular dystrophy

D Duan, N Goemans, S Takeda, E Mercuri… - Nature Reviews …, 2021 - nature.com
Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

Secondary osteoporosis

PR Ebeling, HH Nguyen, J Aleksova… - Endocrine …, 2022 - academic.oup.com
Osteoporosis is a global public health problem, with fractures contributing to significant
morbidity and mortality. Although postmenopausal osteoporosis is most common, up to 30 …

Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis

S Crisafulli, J Sultana, A Fontana, F Salvo… - Orphanet journal of rare …, 2020 - Springer
Abstract Background Duchenne Muscular Dystrophy (DMD) is a rare disorder caused by
mutations in the dystrophin gene. A recent systematic review and meta-analysis of global …

Assessment of systemic delivery of rAAVrh74. MHCK7. micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial

JR Mendell, Z Sahenk, K Lehman, C Nease… - JAMA …, 2020 - jamanetwork.com
Importance Micro-dystrophin gene transfer shows promise for treating patients with
Duchenne muscular dystrophy (DMD) using recombinant adeno-associated virus serotype …

Therapeutic developments for Duchenne muscular dystrophy

IEC Verhaart, A Aartsma-Rus - Nature Reviews Neurology, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Therapeutic approaches for Duchenne muscular dystrophy

TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

Global prevalence of Duchenne and Becker muscular dystrophy: a systematic review and meta-analysis

N Salari, B Fatahi, E Valipour, M Kazeminia… - Journal of orthopaedic …, 2022 - Springer
Background A variety of mutations in the largest human gene, dystrophin, cause a spectrum
from mild to severe dystrophin-associated muscular dystrophies. Duchenne (DMD) and …

Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis

E Landfeldt, R Thompson, T Sejersen… - European journal of …, 2020 - Springer
Several studies indicate that prognosis for survival in Duchenne muscular dystrophy (DMD)
has improved in recent decades. However, published evidence is inconclusive and some …

Emerging therapies for Duchenne muscular dystrophy

T Markati, M Oskoui, MA Farrar, T Duong… - The Lancet …, 2022 - thelancet.com
Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional
dystrophin in the muscle cells. Major advances have led to the development of gene …

Disuse osteoporosis: clinical and mechanistic insights

T Rolvien, M Amling - Calcified tissue international, 2022 - Springer
Disuse osteoporosis describes a state of bone loss due to local skeletal unloading or
systemic immobilization. This review will discuss advances in the field that have shed light …