BioBlast Pharma, a clinical-stage biotechnology firm focused on developing orphan disease drugs, recently announced it has received an allowance from the United States Patent and Trademark Office (USPTO) regarding a method to address oculopharyngeal muscular dystrophy (OPMD) with Cabaletta.
Muscle wasting in muscular dystrophy may be repairable using a new approach developed by a collaboration among researchers in Italy, Israel, and the United Kingdom. Published in EMBO Molecular Medicine, a new study, “In Vivo Generation of a Mature and Functional…
Over 150 advocates associated with the organization Parent Project Muscular Dystrophy (PPMD) gathered on Capitol Hill for PPMD’s Annual Advocacy Conference in the hope of helping accelerate the creation of federal policies to develop and provide treatment options for Duchenne muscular dystrophy (DMD). The meeting between the advocates and more…
One of the largest networks of fertility experts in California, Advanced Reproductive Care (ARC) Fertility, is advocating for the performance of new preimplantation genetic diagnosis (PGD) techniques and preimplantation genetic screening (PGS). The physicians within the network believe the procedures can help more families successfully benefit from fertility treatments and reduce the probability of their unborn…
Biotherapeutics company aTyr Pharma, Inc. was recently awarded orphan drug designation from the European Commission (EC) for its Resolaris therapy, designed to treat facioscapulohumeral muscular dystrophy (FSHD). The investigational drug is the company’s first Physiocrine-based therapy, and is currently being developed to become a first-in-class intravenous protein treatment for…
ReveraGen BioPharma recently announced the start of a Phase 1 clinical trial of a new disassociate steroidal drug called VBP15, indicated to address Duchenne muscular dystrophy (DMD). Recruitment for the trial is currently underway, and the initial doses have already been administered in the first volunteers. “Glucocorticoids are a…
Researchers at Duke University recently revealed an approach to gene therapy that has the potential to treat more than half of the patients with Duchenne Muscular Dystrophy (DMD). The study was published in the journal Nature Communications and is entitled “…
In a recent study entitled “Progression of cardiac involvement in patients with limb-girdle type 2 and Becker muscular dystrophies: A 9-year follow-up study,” researchers describe a higher risk for cardiac dysfunction in patients suffering from Becker muscular dystrophy and limb-girdle muscular dystrophy, particularly subtypes 2I and 2E.
Researchers working in public health have recently published a report based on the first broad study in the United States on the frequency of two common muscle-weakness disorders that mostly affect boys: Duchenne muscular dystrophy and Becker muscular dystrophy. The study was published in …
Britain is now the first country in the world that allows the fusion of DNA from three people for the creation of babies. This could provide a solution for preventing diseases such as muscular dystropy, a hereditary disease characterized by progressive weakening of the muscles. The decision involved a change to in vitro fertilization…
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