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,…
Contributed by Jane Larkindale and Alayna Tress While millions of people globally are living with a rare disease, patients often find it difficult to feel seen or heard throughout their…
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…
In an investor news release from biopharmaceutical company Avidity Biosciences, Inc., the company shared that the FDA placed a partial clinical hold on new enrollment for the Phase 1/2…
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…
According to a recent article in The British publication The Economic Times, a group of researchers at McMaster University have a win-win proposal for myotonic dystrophy (MD) patients. Eleven people were…
Myotonic dystrophy type 2 is a rare and poorly understood condition. Even less understood is its relation to recurrent ventricular tachycardia, which is even rarer. Myotonic Dystrophy Type 2 Myotonic…
According to a story from Rolling Stone, iconic comedian Gilbert Gottfried has passed away from complications related to myotonic dystrophy type II, a rare disease. His publicist disclosed his cause…
This year, the 2022 MDA Clinical & Scientific Conference began March 13, 2022 and is lasting through March 16, 2022. During the conference, stakeholders will discuss a variety of clinical…
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…
As reported recently in Medscape, forty-eight patients with myotonic dystrophy type 1 (DM1), were identified by way of the DM-Scope registry, which is the largest collection of data for the DM population. The…
A recent study has examined what risk factors exist for patients diagnosed with myotonic dystrophy, the most common of all muscular dystrophies. This is a progressive, hereditary condition. Regarding cardiac…
At the end of June 2021, pharmaceutical company Harmony Biosciences Holdings, Inc. ("Harmony") shared the initiation of a Phase 2 clinical trial via news release . During the trial, the…
In many cases, therapies for patients with rare disease are first tested or developed in either 2D cell cultures or in animal models, such as fruit flies, mice, or zebrafish.…
In early January 2021, biopharmaceutical company Avidity Biosciences ("Avidity") announced updates from its 2021 pipeline. Currently, Avidity is working to create a line of Antibody Oligonucleotide Conjugates (AOCs), therapeutic options…
According to an article in BioSpace, Dyne Therapeutics has recently announced data from preclinical trials of its FORCE platform. This program is aimed at creating treatments for myotonic dystrophy type…
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…
According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…
Early this week, biopharmaceutical company Avidity Biosciences announced its inclusion into a collaborative natural history study on myotonic dystrophy type 1 (DM1). The study, END-DM1, is run by the Myotonic…
Last week, biotechnology company Triplet Therapeutics announced their participation in END-DM1, a natural history study to learn more about myotonic dystrophy. There are two major forms of myotonic dystrophy, a rare…
NPR News recently featured an article announcing that scientists at Portland’s Casey Eye Institute have attempted to use CRISPR to edit a gene with the DNA still in place.…
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Stéphanie Tomé is an investigator at the Sorbonne Université in Paris, France. A recent article in PacBio describes Tomé’s research into a disease that becomes progressively worse with each generation.…
A press release from American biopharmaceutical company AMO Pharma, published by PR Newswire, detailed the recent completion of the Company's new congenital myotonic dystrophy type 1 (CDM1) symptom-evaluation scale. The…
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