Category Archives: Muscular Dystrophy Treatment

NEW YORK, Dec. 22, 2020 /PRNewswire/ -- John Howell, Co-Founder and President of the ComSovereign Holding Corporation (Symbol: COMS), the U.S.-based provider of 4G LTE Advanced and 5G-NR (New Radio Systems) communications and technology systems, has been named to the Board of Directors for the Muscular Dystrophy Association (MDA) Continue reading →

LA JOLLA, Calif., Dec. 22, 2020 /PRNewswire/ --Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs), today announced that Sarah Boyce, President and Chief Executive Officer, is scheduled to present at the 39th Annual J.P. Continue reading →

Duchenne muscular dystrophy is a genetic, progressive condition that causes loss of muscle function over time. Its mostly seen in boys and men but can occur, often in a milder form, in girls, and is present at birth. While many new treatments are on the horizon, current treatment options for Duchenne may slow the progression of the disease and treat its symptoms, but cannot halt or reverse its course. Continue reading →

Shareholders of Pfizer (NYSE:PFE) have had an eventful year. A long-awaited spinoff and a global pandemic populated most of the headlines about the company, but those weren't the only developments that will affect the stock in 2021 and beyond. As we approach the holidays, it's useful to look back and ask how much you would have if you had invested in Pfizer at the beginning of the year Continue reading →

Eli Lilly has stepped into the gene therapy space after announcing a deal to acquire Prevail Therapeutics, a company focused on developing adeno-associated virus (AAV)-based gene therapies for neurodegenerative diseases. Lilly will acquire Prevail for $22.50 per share in cash, plus one $4 contingent value right dependent on the first regulatory approval of a product from Prevails pipeline. Continue reading →

CRISPR -- which stands for "clustered regularly interspaced short palindromic repeats" -- has been a hot area of research and investment since scientists discovered that this naturally occurring gene-editing function of bacteria could conceivably be used to treat genetic diseases. Several companies are using gene-editing in their attempts to cure illnesses caused by errors on a single gene such as sickle cell disease, hemophilia, and cystic fibrosis. One of them, CRISPR Therapeutics (NASDAQ:CRSP), has produced results that could not only make it a winner in single-gene disorders, but position it to tackle much more complex -- and profitable -- diseases in the years ahead Continue reading →

Eli Lilly has joined the ranks of big pharma companies building a position in gene therapy, paying $26.50 per share to take control of New York-based Prevail Therapeutics and its two clinical-stage development programs. Prevail has been snapped up just three years after being set up by OrbiMed's co-head of private equity Jonathan Silverstein, aiming to tackle Parkinsons disease (PD) caused by mutations in the glucocerebrosidase (GBA1) genea disease Silverstein was diagnosed with in 2016 Continue reading →

SOUTH PLAINFIELD, N.J., Dec. Continue reading →

December 04, 2020 16:05 ET | Source: Sarepta Therapeutics, Inc. CAMBRIDGE, Mass., Dec Continue reading →

December 07, 2020 08:55 ET | Source: Sarepta Therapeutics, Inc. -- Results from the multiple-ascending dose trial demonstrate proof-of-concept for SRP-5051 and support continued dose escalation -- -- At a total dose exposure approximately 10x lower than eteplirsen, SRP-5051 at 20 mgs/kg showed enhanced tissue exposure, greater exon skipping, and greater dystrophin production with no negative renal or other laboratory findings -- -- These are the first clinical results from the Companys peptide phosphorodiamidate morpholino oligomer (PPMO) technology, its next-generation chemistry platform -- CAMBRIDGE, Mass., Dec. 07, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. Continue reading →