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About 1,963 results

ALLMedicine™ Myotonic Dystrophy Center

Research & Reviews  744 results

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
https://clinicaltrials.gov/ct2/show/NCT03692312

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.

Benefits of aerobic exercise in myotonic dystrophy type 1.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9106338
The Journal of Clinical Investigation; Mackenzie SJ, Hamel J et. al.

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...

Mice lacking MBNL1 and MBNL2 exhibit sudden cardiac death and molecular signatures reca...
https://doi.org/10.1093/hmg/ddac108
Human Molecular Genetics; Lee KY, Seah C et. al.

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...

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
https://clinicaltrials.gov/ct2/show/NCT04886518

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...

2022 HRS expert consensus statement on evaluation and management of arrhythmic risk in ...
https://doi.org/10.1016/j.hrthm.2022.04.022
Heart Rhythm; Groh WJ, Bhakta D et. al.

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...

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Guidelines  3 results

2022 HRS expert consensus statement on evaluation and management of arrhythmic risk in ...
https://doi.org/10.1016/j.hrthm.2022.04.022
Heart Rhythm; Groh WJ, Bhakta D et. al.

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...

Clinical Care Recommendations for Cardiologists Treating Adults With Myotonic Dystrophy.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7070199
Journal of the American Heart Association; McNally EM, Mann DL et. al.

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...

ADDENDUM: Technical standards and guidelines for myotonic dystrophy type 1 testing.
https://doi.org/10.1038/gim.2015.176
Genetics in Medicine : Official Journal of the American C...

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

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Clinicaltrials.gov  23 results

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
https://clinicaltrials.gov/ct2/show/NCT03692312

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.

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
https://clinicaltrials.gov/ct2/show/NCT04886518

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...

DM-IMT - Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1
https://clinicaltrials.gov/ct2/show/NCT04052958

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...

Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1
https://clinicaltrials.gov/ct2/show/NCT04634682

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 ...

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1
https://clinicaltrials.gov/ct2/show/NCT00577577

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...

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News  15 results

Deeper Understanding of NETs Biology May Hold Key to Therapeutic Advances
https://www.onclive.com/view/deeper-understanding-of-nets-biology-may-hold-key-to-therapeutic-advances

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...

Vertex Pharmaceuticals expands into Duchenne gene therapy with new deals
https://www.reuters.com/article/us-exonics-therapeutics-m-a-vertex-pharm/vertex-pharmaceuticals-expands-into-duchenne-gene-therapy-with-new-deals-idUSKCN1T72YK

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...

Blood Test for Alzheimer’s? CTE in Woodpeckers; Comeback for NGF
https://www.medpagetoday.com/neurology/generalneurology/70976

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...

External Resources and Information Related to Patients’ Experience
http://www.fda.gov/drugs/development-approval-process-drugs/external-resources-and-information-related-patients-experience

  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...

External Resources or Information Related to Patients’ Experience
http://www.fda.gov/drugs/development-approval-process-drugs/external-resources-or-information-related-patients-experience

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 ...

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