An estimated 80% or more of people with myotonic dystrophy type 1 (DM1) struggle with severe fatigue and excessive daytime sleepiness (EDS). While most people consider myotonia (inability to contract…
Currently, there are no approved treatment options for myotonic dystrophy type 1 (DM1), a progressive neuromuscular disease. As the disease progresses, affected individuals experience muscle weakness, respiratory distress, and cardiac…
The 2023 Annual Meeting of the Myotonic Dystrophy Foundation took place from September 7-9 this year. During the meeting, stakeholders gathered in the nation's capital to discuss research, trends,…
According to a story from PR Newswire, the biopharmaceutical company AMO Pharma Limited has recently announced the completion of enrollment for its REACH-CDM clinical trial. This trial will be investigating…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Juvena Therapeutics has just announced that they've received a new grant which will fund their research of myotonic dystrophy type 1 (DM1). This was a very competitive grant which totals…
Currently, there are no therapeutic options available for patients with myotonic dystrophy type 1 (DM1). However, that may soon change. According to Marketwatch, the FDA recently granted Fast Track…
Two and a half years after announcing its launch, Dyne Therapeutics held its very first Research & Development Day. Throughout the day, there were presentations focusing on the development of…
According to a story from PR Newswire, the RNA-targeted gene therapy company Locanabio, Inc. has recently released findings from a preclinical evaluation of an RNA-targeted CRISPR Cas9 (RCas9) system as…
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