Duchenne muscular dystrophy
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 …
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 …
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
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 …
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 …
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 …
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 …
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
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 …
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 …
has improved in recent decades. However, published evidence is inconclusive and some …
Emerging therapies for Duchenne muscular dystrophy
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 …
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 …
systemic immobilization. This review will discuss advances in the field that have shed light …