According to a February 25 press release, Amondys 45 (casimersen), a therapeutic option for patients with Duchenne muscular dystrophy (DMD), recently became FDA-approved. The therapy is specifically designed for patients…
According to a story from One News Page, the biopharmaceutical company BridgeBio Pharma and its affiliate ML Bio Solutions have recently announced that dosing has begun in its phase 2…
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…
Solid Biosciences has just reported that the FDA has finally lifted the hold they had placed on their Phase I/II trial for Duchenne muscular dystrophy (DMD). This trial is called…
Parent Project Muscular Dystrophy (PPMD) is a nonprofit that focuses on fighting for a cure, better resources, and more awareness for Duchenne muscular dystrophy (DMD). More than a year ago…
Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…
According to a story from Newswise, trial results released in the journal PLOS Medicine suggest that the drug vamorolone could provide an enhancement in the effectiveness of corticosteroid therapy in patients…
This year, at the 2020 CNS-ICNA Conjoint Meeting, researchers identified ataluren as a potential treatment that inhibits disease progression for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Research suggests…
According to CureDuchenne, two companies have began a collaboration in an effort to create new gene therapies for Duchenne muscular dystrophy (DMD). Ultragenyx and Solid Bioscience are both using their…
While many pharmaceutical companies submit approval and marketing applications, not all are successful. According to Fierce Biotech, a subsidiary of Questex, Santhera Pharmaceuticals ("Santhera"), is one of the most recent…
According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…
At the end of September, Sarepta Therapeutics ("Sarepta") announced positive data from a study exploring SRP-9003, a gene therapy candidate, as a potential treatment for patients with limb-girdle muscular…
The question of whether to go back to school amidst Covid-19 has been a debate across the country. Many universities have seen surges in cases, many involving students in Greek…
As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…
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…
Sarepta Therapeutics has met with the Office of Tissues and Advanced Therapies of the FDA in order to discuss a Type C written response about a clinical trial for SRP-9001,…
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…
According to BioSpace, the first patient has been enrolled in a trial of pamrevlumab, a Duchenne muscular dystrophy treatment. The third phase of LELANTOS plans to evaluate the drug's effects…
As society becomes increasingly technological, researchers are getting closer and closer to mapping the entire human genome. In facts, shares Medical XPress, we may be nearly there. While many…
A team of researchers from the University of Alberta have created a possible treatment for facioscapulohumeral muscular dystrophy (FSHD). Their work has been published in the Proceedings of the National Academy…
When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash…
According to Medical Xpress, a new gene therapy has been studied for the treatment of Duchenne muscular dystrophy. This treatment, SRP-9001, has been shown to deliver micro-dystrophin and improve functional…
By engineering exosomes, Sarepta Therapeutics believes they can utilize gene therapies, RNA therapies, and gene editing without triggering an immune response in the body. They have just announced a new…
In a recent press release, biotechnology company Sarepta Therapeutics announced positive findings from their clinical trial studying SRP-9003 as a treatment for limb-girdle muscular dystrophy (LGMD) type 2E. This…
According to a story from BioPharma Dive, the drug company Sarepta Therapeutics is pushing forward with its plans to develop a gene therapy for limb-girdle muscular dystrophy (LGMD). The company…
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