END-DM1 (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy
Type 1) is a non-interventional study sponsored by the Myotonic Dystrophy
Clinical Research Network (DMCRN), a network of medical centers that aims to support future clinical trials of potential therapies for DM1 through the standardisation of testing methods.
"This is a huge step forward in developing a new approach for treating Duchenne muscular dystrophy
," said Michael Rudnicki of the University of Ottawa.
Duchenne muscular dystrophy
causes muscles to weaken over time, leading to increasing and severe disability.
In literature also mostly reported corneal dystrophies are bilateral.4 According to the French National Waiting List, dystrophies were the third most common indication for penetrating keratoplasty (PKP) and were divided into Fuchs dystrophy
(65%), Lattice dystrophy
(10%), Granular dystrophy
(4%), Macular dystrophy
(2%), and others (20%).5
"The results demonstrate that tamoxifen and raloxifene hold significant potential for treating FKRP-related muscular dystrophy
and probably other muscular dystrophies," the authors write.
LAMA2-related muscular dystrophy
is due to defect in the gene encoding the [alpha]2 subunit, which together with [beta]1 and [gamma]1 chains form laminin-211 (Lm-211), the most prevalent laminin found in basement membrane (BM), surrounding muscle fibers, and also in the Schwann cells of peripheral nerves .
All patients received standard-of-care for Duchenne muscular dystrophy
, including oral steroids, and 13 also received one dose of intracoronary CAP-1002 upon randomization.
The differential diagnosis of this condition included central areolar choroidal dystrophy
, geographic atrophy, and pathologic myopia.
Biopharmaceutical company AMO Pharma Limited revealed on Thursday the receipt of the US Food and Drug Administration's (FDA) orphan drug designation for its investigational therapy in development AMO-02 for the treatment of congenital myotonic dystrophy
Key Words: Muscle dystrophy
, Dysmorphic face, Jaw muscle weakness.
The "Move a Mile for Muscles" walk was organised by Kate Cartwright of Aberdare, whose son Sonny, six, has the muscle wasting condition Becker muscular dystrophy