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…
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…
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…
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…
Clinical trials are important to understanding both more about certain conditions and more about the safety, efficacy, and tolerability of drugs designed to treat these conditions. Currently, biopharmaceutical company AMO…
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…
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 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…
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…
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