Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told BioNews Services, publisher…

The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the most determined. At that time, few doctors in the developing country of 2 million — now called North Macedonia — had…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies(NCATS) at the…

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

Under a restructured agreement with Regulus Therapeutics, Sanofi Genzyme now has sole responsibility for the development and marketing of the investigational therapy RG-012, currently under evaluation for the treatment of Alport syndrome. The amendment to the prior collaboration agreement between Sanofi and Regulus, RG-012’s initial developer, gives…

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

Regulus Therapeutics and Sanofi-Genzyme are among companies working on Alport syndrome therapies that can block RNA molecules’ actions. RG-012 is a man-made version of naturally occurring oligonucleotides, or RNA molecules that help regulate gene activity. The synthetic version, called antisense oligonucleotides, binds to RNA, preventing it from functioning normally. But what are…