According to ASHA.org, when Tiffany Turner graduated, she set out to expand opportunities for fellow speech language pathologists like herself. Turner saw a great need in her community for…
According to News Medical, a team of researchers from London have created new technologies that may aid in further understanding Duchenne muscular dystrophy (DMD). Not only this, but the new…
According to a story from PR Newswire, the specialty drug development company Mallinckrodt recently announced that enrollment for trials of its experimental medication MNK-1411 has started. The drug will be…
According to a story from the Los Angeles Daily News, for children with debilitating rare illnesses like cerebral palsy, epilepsy, or muscular dystrophy, it can often take a long time…
An experimental gene therapy program (called AXO-AAV-OPMD) being developed for the treatment of oculopharyngeal muscular dystrophy has been licensed with exclusive global rights to Axovant Sciences from Benitec Biopharma. There…
According to a July 9 report by the Muscular Dystrophy Association (MDA), the MDA awarded a large grant to researchers studying Charcot-Marie-Tooth disease. The grant, totaling over $1,000,000, was awarded…
According to a story from BioSpace, the biopharmaceutical company Acceleron Pharma, which develops TGF-beta based treatments for rare and serious illnesses, recently announced that the U.S. Food and Drug Administration…
According to BioPortfolio, the company UniQure has recently announced that the first hemophilia B patient has been enrolled in their Phase III study of the treatment AMT-061, which is an…
According to UVA Today, a University of Virginia professor, Sarah Kucenas, is studying glia cells in the brain of fish that could potentially help spear the way for the creation of…
Many people are familiar with Carl Zimmer as a science columnist from The New York Times. His columns and research dug deeply into the field of genetic research and gene…
According to Bloomberg, there’s a new big fish in the medical technology pond. Crispr Therapeutics AG jumped in value, multiplied by six, and landed at $3 billion. How is that…
According to a story from apnews.com, President Trump signed the "Right to Try" bill passed by Congress into law on Wednesday, May 30th, 2018. During the signing ceremony, he was…
According to a story from Business Wire, the gene therapy company Myonexus Therapeutics announced that the U.S. FDA has given Rare Pediatric Disease Designation for its gene therapy candidate MYO-101.…
The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug Sarconeos, developed by the company Biophytis, for the treatment of Duchenne muscular dystrophy. The…
According to a story from wlwt.com, thirteen-year-old Diego Ramirez has been crossing the finish line at the Flying Pig Marathon for seven years, and he recently completed the finish again…
Acceleron Pharma has just received Fast Track designation from the US Food and Drug Administration (FDA) for their experimental treatment ACE-083 designed to treat patients with facioscapulohumeral muscular dystrophy (FSHD).…
Phillippa Farrant, whose son, Daniel, has been diagnosed with Duchenne muscular dystrophy (DMD), recently appeared in Parliament in the UK to fight for improved access to care and treatment for…
The phrase “it takes a village” gets tossed around a lot. But in Codicote, that’s what’s happening. Fundraising events and challenges are being held left and right. All of them…
Researchers at the University of Hong Kong have discovered that a drug used to treat muscular dystrophy also works on a hereditary heart disease called Lamin A/C-related dilated cardiomyopathy. The…
Pfizer has just begun a medical trial for a potential gene therapy treatment for Duchenne muscular dystrophy, and the first patient has been dosed. Click here to read the original…
There is a growing consensus that patients need to be more included in the medical field, reports PCMA Convene. Over the last ten years increasing numbers of organisations have moved…
According to a story from genengnews.com, the pharmaceutical company Solid Biosciences is pledging to continue developing its investigation drug candidate SGT-001 for Duchenne muscular dystrophy. This is despite its clinical…
Clinical trials of a potential gene therapy treatment for Duchenne muscular dystrophy (DMD) have been put on hold after concerns emerged about the safety of the procedure, reports Genetic Engineering…
According to a story from Business Wire, there was a time when having to use a mechanical ventilator in order to breathe meant being confined at the hospital in an…
According to a story from financialbuzz.com, the biotechnology company BIOPHYTIS has submitted documentation for orphan drug designation from the European Medicines Agency (EMA). This designation will be for Sarconeos, a…
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