News

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

The investigational therapy edasalonexent was seen to slow disease progression in boys with Duchenne muscular dystrophy, almost one-year results from the MoveDMD trial report. Specifically, edasalonexent — administered orally as 100 mg/kg dose for 48 weeks — resulted in a statistically significant delay in disease progression and signs of…

The U.S. Food and Drug Administration (FDA) granted orphan drug status to MYO-101, a gene therapy being developed by Myonexus Therapeutics for the treatment of limb girdle muscular dystrophy type 2E (LGMD2E) . The FDA’s orphan drug designation is recognition of the potential of MYO-101 for the treatment of…

A national survey to identify how Americans with disabilities are coping with work and overcoming barriers to employment has found that, despite persistent challenges, their efforts are making a big difference. The 2015 Kessler Foundation National Employment and Disability Survey (KFNEDS) was conducted by a multidisciplinary team of researchers at…

It’s not every day a Southeast Asian monarchy bestows a $100,000 prize on a U.S. government research agency. In fact, it rarely happens. But this year is different. Thailand’s Prince Mahidol Award Foundation has chosen the National Human Genome Research Institute — a division of the National Institutes of Health (NIH)…

A potential next-generation therapy, golodirsen (SRP-4053) facilitates and increases dystrophin production in Duchenne muscular dystrophy (DMD) patients, an interim analysis of a Phase 1/2 trial being presented at the 2018 American Academy of Neurology (AAN) Annual Meeting in Los Angeles reports. These findings, in  “Golodirsen Induces Exon Skipping Leading…

Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) than standard of care corticosteroid treatments, according to a re-analysis of data from two clinical trials. The new findings follow a reassessment of pooled data obtained in the placebo group…

Treatment with Exondys 51 (eteplirsen) was found to be safe and well-tolerated in a very young infant with Duchenne muscular dystrophy (DMD), preliminary data of a follow-up study shows. Initial results of the study will be the subject of a poster presentation at the 2018 Annual…

Eric Olson, a molecular biologist, has spent more than half his life in the lab. Now, the 62-year-old University of Texas researcher and distinguished professor is a businessman as well. Olson, director of UT Southwestern’s Hamon Center for Regenerative Science and Medicine in Dallas, has teamed up with…

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…