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We are pleased to invite investors and analysts to participate in our virtual event on Friday, 19 March, 2021, highlighting Roche data presented during the 2021 Muscular Dystrophy Association - MDA -Virtual Clinical and Scientific Conference, from 15th to 18th February. (Source: Roche Investor Update)
Courtney Young helped develop the gene therapy at the heart of a biotech startup,MyoGene Bio, when she was a doctoral student at UCLA from 2013 to 2018. But the kernel of the company ’s endeavor is older still — and definitively personal.In 2008, when Young was in high school, she found out that her cousin, then just a toddler, had been diagnosed with Duchenne muscular dystrophy. She made it her mission to cultivate the skills needed to provide better options for him and others who were suffering with the deadly muscle-wasting disease.“I directed my career toward working on Duchenne,” said Young, MyoGene Bio’s co-founder and CEO. “I came to UCLA specifically because there’s a high concentration of researchers working on the disease. My advisers and I decided to develop a nove...
Hailed as the ‘molecular scissors’ that will allow us to rewrite our genes, the DNA tool is being trialled in treatments for everything from sickle-cell anaemia to cancerWhen last year ’s Nobel prize for chemistry was awarded tobiochemist Jennifer Doudna and microbiologist Emmanuelle Charpentier for their work in developing the technique of gene editing known as Crispr-Cas9 (pronounced “crisper”), headlines hailed their discovery as “molecular scissors” that would allow us to “rewrite the book of life” – with all the complicated ethical questions that ability raises. But much of the excitement has nothing to do with visions of designer babies. The real promise of Cri spr is for treating diseases caused by genetic mutations, from muscular dystrophy to congenital blindnes...
The FDA has approved casimersen (Amondys 45) injection for treating patients with both Duchenne muscular dystrophy (DMD) and a rare mutation of the DMD gene.FDA Approvals (Source: Medscape Pediatrics Headlines)<div id="medworm"><p><b><i>MedWorm Message:</i></b> Have you tried our new <a href="https://medworm.com/">medical search engine</a>? More powerful than before. Log on with your social media account. 100% free.</p></div>
CAMBRIDGE, Mass., Feb. 25, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Amondys 45... (Source: Drugs.com - New Drug Approvals)