Category Archives: Muscular Dystrophy Treatment

Pratteln, Switzerland, January27, 2020 Santhera Pharmaceuticals (SIX: SANN) generated net revenues of CHF27.9 million in 2019 from sales of Raxone for the treatment of Lebers hereditary optic neuropathy (LHON), which was out-licensed to Chiesi Group from August 2019. The Company made strong progress in advancing its lead neuromuscular compounds Puldysa (idebenone) and vamorolone towards market entry and regulatory submission, respectively. We are pleased about Santheras strong progress in 2019 and are excited about the prospects 2020 holds, said Dario Eklund, CEO of Santhera Continue reading →

Containing new coronavirus may not be feasible, experts say, Containing new coronavirus may not be feasible, experts say, as they warn of possible sustained global DNA sleuths read the coronavirus genome, tracing its origins DNA sleuths read the coronavirus genome, tracing its origins and looking for dangerous mutations How fast can biotech come up with a vaccine How fast can biotech come up with a vaccine for the latest outbreak? Continue reading →

The discovery of new viruses, vaccines, and treatments in 2019 was the result of investments in global surveillance, cross-sector partnerships, and scientific advances. At a time when misinformation is calling into question the validity of facts, the world needs to support science more than ever. ZURICH Measles went viral in 2018, infecting nearly ten million people worldwide and claiming 142,000 lives Continue reading →

Sarepta Therapeutics (SRPT) fell victim to the FDA's whims as the stock tumbled more than 3 percent following the issues raised by the FDA against its drug candidate Vyondys 53 (golodirsen). The drug was earlier rejected by the FDA last year. Continue reading →

The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to Sarepta Therapeutics in August 2019 over its golodirsen (Vyondys 53) for the treatment of Duchenne muscular dystrophy (DMD) Continue reading →

Former chief executive officer of Myotonic brings extensive experience in patient advocacy and community engagement Dyne Therapeutics, a biotechnology company pioneering targeted therapies for patients with serious muscle diseases, today announced the appointment of Molly White as Vice President, Medical Communications and Advocacy. Continue reading →

A biotech startup in La Jolla has raised $85 million from notable science investors to investigate its idea for treating age-related diseases: a pill that mimics the effects of exercise and fasting. The company, called Epirium Bio, says its scientists have discovered a new human hormone, which, when influenced, can trigger the production of more mitochondria Continue reading →

CAMBRIDGE, Mass., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (SRPT), the leader in precision genetic medicine for rare diseases, announced that it has appointed John C Continue reading →

Given his level of education, the resources at his disposal and his media celebrity, one would place Montel Williams near the bottom of any list of individuals likely to endure a missed diagnosis for two full decades. And yet there he was during the bulk of the 1980s and 90s, enduring pain in his lower extremities and temporary blindness in one eye as doctor after doctor failed to diagnose him with relapsing-remitting multiple sclerosis. That he lived like this while, among other things, serving in the Navy, hosting a syndicated TV talk show and becoming a father to four children, ranks as a fairly amazing achievement in retrospect. Continue reading →

By: Janet Woodcock, M.D., Director, Center for Drug Evaluation and Research Happy New Year! The beginning of a new year is always a rewarding time for the many dedicated staff at the FDA's Center for Drug Evaluation and Research (CDER) who work on approving new drug therapies. We look back over the past year and see how the work we did came together to help patients in need one of the main reasons why many of us, including me, have chosen a career in regulatory science and public health. Continue reading →