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30 results on '"Myotonic dystrophy -- Drug therapy"'

1. Delpacibart Etedesiran Gets Breakthrough Tx Status for Myotonic Dystrophy Type 1

Author

Ernst, Diana

Subjects
United States. Food and Drug Administration, Drug therapy, Myotonic dystrophy -- Drug therapy
Abstract

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001) for the treatment of myotonic dystrophy type 1 (DM1). https://www.empr.com/home/news/drugs-in-the-pipeline/oligonucleotide-therapeutic-gets-fast-track-status-for-myotonic-dystrophy-type-1/ is a rare genetic [...]

Published
2024

2. Arrakis Therapeutics Unveils Data for its Lead, Rationally Designed, RNA-Targeted Small Molecule (rSM) Drug Program Demonstrating Disease-Modifying Binding to RNA to Treat Myotonic Dystrophy

Subjects
Diseases -- Drug therapy, Drug discovery, Drugs, RNA, Myotonic dystrophy -- Drug therapy, General interest, News, opinion and commentary
Abstract

WALTHAM: ARRAKIS THERAPEUTICS has issued the following news release: Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today [...]

Published
2024

3. Arrakis Therapeutics Unveils Data for its Lead, Rationally Designed, RNA-Targeted Small Molecule (rSM) Drug Program Demonstrating Disease-Modifying Binding to RNA to Treat Myotonic Dystrophy

Subjects
Diseases -- Drug therapy -- Massachusetts, Drugs -- Product development, RNA, Myotonic dystrophy -- Drug therapy, Business, Business, international
Abstract

Preclinical data presented at Cell Symposia conference show a small molecule directed at a pathogenic RNA achieved reversal of myotonia in an animal model of myotonic dystrophy Type 1 (DM1) [...]

Published
2024

4. Juvena Receives FDA Orphan Drug Designation for JUV-161 for the Treatment of Myotonic Dystrophy Type 1

Subjects
United States. Food and Drug Administration, Stem cells -- Physiological aspects, Orphan drugs -- Physiological aspects, Proteins -- Physiological aspects, Myotonic dystrophy -- Drug therapy, Arts and entertainment industries
Abstract

Juvena Therapeutics, a biotechnology company unlocking the potential of stem cell-secreted proteins to treat muscle and metabolic diseases including muscular dystrophies and obesity, reported the U.S. Food and Drug Administration [...]

Published
2024

6. PepGen Receives U.S. FDA Fast Track Designation for PGN-EDODM1 for the Treatment of Myotonic Dystrophy Type 1

Subjects
United States. Food and Drug Administration, Myotonic dystrophy -- Drug therapy, Banking, finance and accounting industries, Business
Abstract

BOSTON, Feb. 20, 2024 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of [...]

Published
2024

7. Juvena Therapeutics Receives FDA Orphan Drug Designation for JUV-161 for the Treatment of Myotonic Dystrophy Type 1

Subjects
United States. Food and Drug Administration, Stem cells, Orphan drugs, Proteins, Myotonic dystrophy -- Drug therapy, Business, Business, international
Abstract

Biologic treatment that restores muscle fiber formation leads Juvena's pipeline of assets to treat myopathies, metabolic diseases and obesity REDWOOD CITY, Calif. -- Juvena Therapeutics, a biotechnology company unlocking the [...]

Published
2024

8. Juvena Gets FDA Orphan Drug Designation for JUV-161 for the Treatment of Myotonic Dystrophy Type 1

Subjects
United States. Food and Drug Administration, Stem cells -- Physiological aspects, Orphan drugs -- Physiological aspects, Proteins -- Physiological aspects, Myotonic dystrophy -- Drug therapy, Business, Business, international, Telecommunications industry
Abstract

Juvena Therapeutics, a biotechnology company unlocking the potential of stem cell-secreted proteins to treat muscle and metabolic diseases including muscular dystrophies and obesity, reported the U.S. Food and Drug Administration [...]

Published
2024

9. Investigational Therapy for Myotonic Dystrophy Type 1 Gets Fast Track Status

Author

Park, Brian

Subjects
United States. Food and Drug Administration, Drug therapy, Myotonic dystrophy -- Drug therapy, Monoclonal antibodies, RNA
Abstract

The Food and Drug Administration (FDA) has granted Fast Track designation to AOC 1001 for the treatment of myotonic dystrophy type 1 (DM1). DM1 is a rare genetic disorder caused [...]

Published
2021

10. Study Data from Institute for Cancer Research and Treatment (IRCCS) Foundation Santa Lucia Update Understanding of Myotonic Dystrophy (Ventral tegmental area dysfunction affects decision-making in patients with myotonic dystrophy type-1)

Subjects
Drug therapy, Myotonic dystrophy -- Drug therapy, Physical fitness, Cancer -- Drug therapy, Cancer research, Cognitive disorders, Obesity, Finance, Editors, Diseases
Abstract

2020 MAY 23 (NewsRx) -- By a News Reporter-Staff News Editor at Obesity, Fitness & Wellness Week -- Investigators publish new report on Musculoskeletal Diseases and Conditions - Myotonic Dystrophy. [...]

Published
2020

11. FDA Grants Fast Track Designation to AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Subjects
United States. Food and Drug Administration, Biological products, Myotonic dystrophy -- Drug therapy, General interest, News, opinion and commentary
Abstract

LA JOLLA: Avidity Biosciences, Inc. has issued the following news release: Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody [...]

Published
2021

12. FDA Grants Fast Track Designation to AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Subjects
United States. Food and Drug Administration, RNA, Myotonic dystrophy -- Drug therapy, Business, News, opinion and commentary
Abstract

LA JOLLA, Calif., Oct. 18, 2021 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs(TM)), [...]

Published
2021

13. Creatine monohydrate in myotonic dystrophy; A double-blind, placebo-controlled clinical study

Author

Walter, Maggie C., Reilich, Peter, Lochmuller, Hanns, Kohnen, Ralph, Schlotter, Beate, Hautmann, Hubert, Dunkl, Elmar, Pongratz, Dieter, and Muller-Felber, Wolfgang

Subjects
Myotonic dystrophy -- Drug therapy, Myotonic dystrophy -- Research, Creatine -- Dosage and administration, Health
Abstract

Byline: Maggie C. Walter (1), Peter Reilich (1), Hanns Lochmuller (1), Ralph Kohnen (2), Beate Schlotter (1), Hubert Hautmann (3), Elmar Dunkl (2), Dieter Pongratz (1), Wolfgang Muller-Felber (1) Keywords: Key words myotonic dystrophy (MD); creatine monohydrate; therapy; supplementation Abstract: We assessed safety and efficacy of creatine monohydrate (Cr) in myotonic dystrophy (DM1) in a double-blind, cross-over trial. Thirty-four patients with defined DM1 were randomized to receive Cr and placebo for eight weeks (10.6 g day 1--10, 5.3 g day 11--56) in one of 2 treatment sequences. There was no significant improvement using manual and quantitative muscle strength, daily-life activities, and patients' own global assessment comparing verum with placebo administration. Cr supplementation was well tolerated without clinically relevant side effects, but did not result in significant improvement of muscle strength or daily-life activities. Author Affiliation: (1) Friedrich-Baur-Institute, Dept. of Neurology, Ludwig-Maximilians-University, Ziemssenstr. 1a, 80336 Munich, Germany. Maggie.Walter@lrz.uni-muenchen.de, DE (2) IMEREM Institute for Medical Research Management and Biometrics, Nurnberg, Germany, DE (3) Friedrich-Baur-Institute, Department of Pulmonology, Ludwig-Maximilians-University, Munich, Germany, DE Article note: Received: 1 May 2002, Received in revised form: 5 July 2002, Accepted: 12 July 2002 Correspondence to Dr. M. C. Walter

Published
2002

15. AMO Pharma to Participate in Myotonic Dystrophy Foundation 'Meet the DM Drug Developers' Program on March 5, 2021

Subjects
Medical research, Medicine, Experimental, Genetic disorders -- Drug therapy, Myotonic dystrophy -- Drug therapy, Business, News, opinion and commentary
Abstract

Company to present update on REACH-CDM pivotal clinical trial studying use of AMO-02 in treatment of congenital myotonic dystrophy LONDON, March 3, 2021 /PRNewswire/ -- AMO Pharma Limited ('AMO Pharma'), [...]

Published
2021

16. FDA Grants Rare Pediatric Disease Designation to AMO Pharma for AMO-02 for Treatment of Congenital Myotonic Dystrophy

Subjects
United States. Food and Drug Administration, Children -- Diseases, Genetic disorders -- Drug therapy, Myotonic dystrophy -- Drug therapy, Business, News, opinion and commentary
Abstract

Designation qualifies AMO-02 for fast track review and AMO Pharma to receive a priority review voucher pending marketing approval. LONDON, Nov. 9, 2020 /PRNewswire/ -- AMO Pharma Limited ('AMO Pharma'), [...]

Published
2020

17. US FDA grants orphan drug status to AMO Pharma's congenital myotonic dystrophy drug, AMO-02

Subjects
United States. Food and Drug Administration, Drug therapy, Product development, Myotonic dystrophy -- Drug therapy, Orphan drugs -- Product development, Genetic disorders -- Drug therapy
Abstract

AMO Pharma Limited, a privately held biopharmaceutical company, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for AMO-02, the company's investigational therapy in development [...]

Published
2017

18. Institute for Cancer Research and Treatment (IRCCS) Details Findings in Myotonic Dystrophy (Look at the cognitive deficits in patients with myotonic dystrophy type 1)

Subjects
Drug therapy, Reports, Cancer treatment -- Reports, Myotonic dystrophy -- Drug therapy, Cancer -- Drug therapy, Cancer research -- Reports, Editors, Diseases
Abstract

2020 MAY 29 (NewsRx) -- By a News Reporter-Staff News Editor at Health & Medicine Week -- Fresh data on Musculoskeletal Diseases and Conditions - Myotonic Dystrophy are presented in [...]

Published
2020

19. Cancer drug shows promise in reducing toxic genetic material in myotonic dystrophy

Subjects
Drug therapy, Myotonic dystrophy -- Drug therapy, Genetic research, Cancer -- Drug therapy, Antineoplastic agents, RNA
Abstract

2016 JAN 2 (NewsRx) -- By a News Reporter-Staff News Editor at Obesity, Fitness & Wellness Week -- GAINESVILLE, Fla. -- A group of researchers has shown for the first [...]

Published
2016

20. AMO Pharma Receives FDA Orphan Drug Designation for AMO-02 for Treatment of Congenital Myotonic Dystrophy

Subjects
United States. Food and Drug Administration, Myotonic dystrophy -- Drug therapy, Orphan drugs, Genetic disorders -- Drug therapy, General interest, News, opinion and commentary
Abstract

LONDON: AMO Pharma Limited has issued the following news release: AMO Pharma Limited ('AMO Pharma'), a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, [...]

Published
2017

21. AMO Pharma wins US FDA's orphan drug designation for AMO-02 for treatment of congenital myotonic dystrophy

Subjects
United States. Food and Drug Administration, Myotonic dystrophy -- Drug therapy, Orphan drugs -- Product development, Pharmaceutical industry -- Product development, Genetic disorders -- Drug therapy, General interest, News, opinion and commentary
Abstract

Jul 28, 2017 (M2 EQUITYBITES via COMTEX) -- Biopharmaceutical company AMO Pharma Limited revealed on Thursday the receipt of the US Food and Drug Administration's (FDA) orphan drug designation for [...]

Published
2017

22. AMO Pharma Receives FDA Orphan Drug Designation for AMO-02 for Treatment of Congenital Myotonic Dystrophy

Subjects
United States. Food and Drug Administration, Genetic disorders -- Drug therapy, Orphan drugs -- Product development, Myotonic dystrophy -- Drug therapy, Business, News, opinion and commentary
Abstract

LONDON and PHILADELPHIA, July 27, 2017 /PRNewswire/ -- AMO Pharma Limited ('AMO Pharma'), a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, today announced [...]

Published
2017

23. AMO Pharma Announces FDA Fast Track Designation For AMO-02 For Treatment Of Congenital Myotonic Dystrophy

Subjects
United States. Food and Drug Administration, Genetic disorders -- Drug therapy, Myotonic dystrophy -- Drug therapy, Business, News, opinion and commentary
Abstract

LONDON and PHILADELPHIA, May 30, 2017 /PRNewswire/ -- AMO Pharma Limited ('AMO Pharma'), a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, today announced [...]

Published
2017

24. New Myotonic Dystrophy Study Findings Recently Were Reported by Researchers at Institute for Cancer Research and Treatment (IRCCS) (Neuropsychological and Psychological Functioning Aspects in Myotonic Dystrophy Type 1 Patients in Italy)

Subjects
Drug therapy, Cancer treatment, Mental disorders -- Drug therapy, Myotonic dystrophy -- Drug therapy, Cancer -- Drug therapy, Cancer research, Mental health
Abstract

2018 OCT 22 (NewsRx) -- By a News Reporter-Staff News Editor at Mental Health Weekly Digest -- New research on Musculoskeletal Diseases and Conditions - Myotonic Dystrophy is the subject [...]

Published
2018

25. AMO Pharma wins US FDA's orphan drug designation for AMO-02 for treatment of congenital myotonic dystrophy

Subjects
United States. Food and Drug Administration, Myotonic dystrophy -- Drug therapy, Orphan drugs -- Product development, Pharmaceutical industry -- Product development, Genetic disorders -- Drug therapy, Chemistry
Abstract

M2 PHARMA-July 28, 2017-AMO Pharma wins US FDA's orphan drug designation for AMO-02 for treatment of congenital myotonic dystrophy (C)2017 M2 COMMUNICATIONS Biopharmaceutical company AMO Pharma Limited revealed on Thursday [...]

Published
2017

26. AMO Pharma wins US FDA's orphan drug designation for AMO-02 for treatment of congenital myotonic dystrophy

Subjects
United States. Food and Drug Administration, Myotonic dystrophy -- Drug therapy, Orphan drugs -- Product development, Pharmaceutical industry -- Product development, Genetic disorders -- Drug therapy, Business
Abstract

M2 EQUITYBITES-July 28, 2017-AMO Pharma wins US FDA's orphan drug designation for AMO-02 for treatment of congenital myotonic dystrophy (C)2017 M2 COMMUNICATIONS http://www.m2.com Biopharmaceutical company AMO Pharma Limited revealed on [...]

Published
2017

27. AMO Pharma granted Fast Track designation by US FDA for potential treatment for congenital myotonic dystrophy

Subjects
United States. Food and Drug Administration, Myotonic dystrophy -- Drug therapy, Drug approval, Genetic disorders -- Drug therapy, Business
Abstract

M2 EQUITYBITES-May 30, 2017-AMO Pharma granted Fast Track designation by US FDA for potential treatment for congenital myotonic dystrophy (C)2017 M2 COMMUNICATIONS http://www.m2.com Privately owned biopharmaceutical company AMO Pharma Ltd [...]

Published
2017

28. AMO Pharma granted Fast Track designation by US FDA for potential treatment for congenital myotonic dystrophy

Subjects
United States. Food and Drug Administration, Myotonic dystrophy -- Drug therapy, Drug approval, Genetic disorders -- Drug therapy, Chemistry
Abstract

M2 PHARMA-May 30, 2017-AMO Pharma granted Fast Track designation by US FDA for potential treatment for congenital myotonic dystrophy (C)2017 M2 COMMUNICATIONS Privately owned biopharmaceutical company AMO Pharma Ltd has [...]

Published
2017

29. Reports Summarize Myotonic Dystrophy Findings from Institute for Cancer Research and Treatment (IRCCS) (Validation of plasma microRNAs as biomarkers for myotonic dystrophy type 1)

Subjects
Drug therapy, Cancer treatment, MicroRNA, Myotonic dystrophy -- Drug therapy, Cancer -- Drug therapy, Cancer research, Biological markers
Abstract

2016 DEC 30 (NewsRx) -- By a News Reporter-Staff News Editor at Health & Medicine Week -- Investigators discuss new findings in Musculoskeletal Diseases and Conditions - Myotonic Dystrophy. According [...]

Published
2016

30. Modafinil for Myotonic Dystrophy

Author

RUBIN, MICHAEL

Subjects
Modafinil -- Health aspects, Myotonic dystrophy -- Drug therapy, Health, Psychology and mental health
Abstract

Source: Damian MS, et al. Modafinil for excessive daytime sleepiness in myotonic dystrophy. Neurology. 2001;56: 794-796. HYPERSOMNIA IS A PROMINENT FEATURE OF MYOTONic dystrophy (MyD), although, interestingly, it is more [...]

Published
2001

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