Utrophin upregulation for treating Duchenne or Becker muscular dystrophy
: how close are we?
muscular dystrophy Becker muscular dystrophy
Limb-girdle muscular dystrophy Facioscapulohumeral
Prevalence and incidence of Becker muscular dystrophy
. Lancet 1991;337:1022-4.
This report provides comprehensive information on the therapeutic development for Becker Muscular Dystrophy
, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases.
The structure of the N-terminal actin-binding domain of human dystrophin and how mutations in this domain may cause Duchenne or Becker muscular dystrophy
. Structure 2000; 8:481-491.
[ClickPress, Fri Mar 27 2015] GlobalData's clinical trial report, "Becker Muscular Dystrophy
Global Clinical Trials Review, H1, 2015" provides data on the Becker Muscular Dystrophy
clinical trial scenario.
Becker muscular dystrophy
(BMD) (OMIM #300376) is a milder form of the disease, caused by mutations in the same gene.
In a clinical trial, tadalafil--commonly known by brand names Cialis and Adcirca--reversed the effects of a biochemical chain of events that, in Becker muscular dystrophy
, deprives muscles of an important chemical, nitric oxide, which normally tells blood vessels to relax during exercise, increasing blood flow and oxygenation.
Of note, he previously had genetic testing for Becker muscular dystrophy
(based on his initial presentation at age 4.5 of proximal muscle weakness), which revealed a variant of unknown clinical significance.
Molecular genetic analysis of 67 patients with Duchenne/ Becker muscular dystrophy
. Hum Genet 1992;90: 65-70.
Steve Higginson, 49, from Colwyn Bay, has struggled to find appropriate care for his Becker muscular dystrophy
for many years.
Mutations in the dystrophin (Duchenne muscular dystrophy [ DMD ]) gene, which encodes a protein connecting the cytoskeleton of muscle fibers, result in X-linked recessive dystrophinopathy, including DMD and Becker muscular dystrophy
(BMD).[sup] DMD is the most common type of muscular dystrophy, affects 1:3500 to 6000 live male births, and is characterized by weakness of pelvic and shoulder muscles starting in early childhood.[sup] DMD is thought to be caused by the mutations causing totally nonfunctional dystrophin protein.[sup] In comparison, a reduced amount or shortened dystrophin was thought to lead in BMD, which has a milder clinical manifestation and better prognosis.