ALLMedicine™ Myotonic Dystrophy Center

May 17th, 2022 - Myotonic dystrophy type 1 (DM1) is a multisystem trinucleotide repeat expansion disorder characterized by the misregulated alternative splicing of critical mRNAs. Previous work in a transgenic mouse model indicated that aerobic exercise effectivel...

May 17th, 2022 - This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.

May 15th, 2022 - Myotonic dystrophy (DM) is caused by expansions of C(C)TG repeats in the non-coding regions of the DMPK and CNBP genes, and DM patients often suffer from sudden cardiac death due to lethal conduction block or arrhythmia. Specific molecular changes...

May 5th, 2022 - The study will consist of a Screening Period, an 11-week Double-Blind Treatment Phase (including a 3-week Titration Period and an 8-week Stable Dose Period), and an optional Open Label Extension (OLE) Phase. The OLE Phase will last approximately o...

May 3rd, 2022 - This international multidisciplinary document is intended to guide electrophysiologists, cardiologists, other clinicians, and health care professionals in caring for patients with arrhythmic complications of neuromuscular disorders (NMDs). The doc...

May 3rd, 2022 - This international multidisciplinary document is intended to guide electrophysiologists, cardiologists, other clinicians, and health care professionals in caring for patients with arrhythmic complications of neuromuscular disorders (NMDs). The doc...

Feb 19th, 2020 - Myotonic dystrophy is an inherited systemic disorder affecting skeletal muscle and the heart. Genetic testing for myotonic dystrophy is diagnostic and identifies those at risk for cardiac complications. The 2 major genetic forms of myotonic dystro...

Jan 13th, 2016 - ADDENDUM: Technical standards and guidelines for myotonic dystrophy type 1 testing.|2016||diagnosis,genetics,

May 17th, 2022 - This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.

May 5th, 2022 - The study will consist of a Screening Period, an 11-week Double-Blind Treatment Phase (including a 3-week Titration Period and an 8-week Stable Dose Period), and an optional Open Label Extension (OLE) Phase. The OLE Phase will last approximately o...

May 2nd, 2022 - For some diseases, regular respiratory muscle training could delay the start of ventilation. For DM1, however, there are no clinically high-quality studies. Only a case description from the year 2006 showed a missing improvement of the symptoms af...

Apr 6th, 2022 - Detailed Description: Myotonic Dystrophy type I (DM1) is the most common form of adult muscular dystrophy, affecting 1 in 8000 individuals. It is an autosomal dominant disorder with multisystemic involvement of multiple organs and tissues, mainly ...

Jan 6th, 2022 - Efficacy Measures: Endurance, Ambulation, Cognitive function, Insulin resistance, Cholesterol and triglycerides, Muscle function and strength, Pain, Gastrointestinal function, Quality of life MINIMUM INCLUSION CRITERIA A diagnosis of DM1, confirme...

Oct 6th, 2021 - Lakshmi N. Rajdev, MD As a greater understanding of the biological differences across neuroendocrine tumor (NET) subtypes is observed, other therapeutic strategies beyond somatostatin analogs are coming to the forefront, said Lakshmi N. Rajdev, M...

Jun 6th, 2019 - (Reuters) - Vertex Pharmaceuticals Inc said here on Thursday it would acquire privately held Exonics Therapeutics for an upfront payment of $245 million and potential milestone payments of up to $750 million. Exonics Therapeutics develops gene edi...

Feb 6th, 2018 - A team of Australian and Japanese scientists announced a novel blood test to detect amyloid-β buildup in the brain. Measurements of the protein and its precursors in blood can predict amyloid-β deposition, paving the way for an efficient screening...

  This webpage is intended to facilitate public discussion of patient-focused drug development and evaluation. This webpage provides links to certain publicly available external reports and resources relating to patient experience data. The patien...

This webpage is intended to facilitate public discussion of patient-focused drug development and evaluation. This webpage provides links to certain publicly available external reports and resources relating to patient experience data. The patient ...