Author Archives: Larry Luxner

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise that people with rare diseases — whose immune systems were generally compromised even before the outbreak — are particularly terrified of this pandemic. That fear is evident in an online survey conducted by the National…

Gene Therapy Takes Center Stage at 2019 NORD Summit

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

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…

European Initiative Targets Diagnosis, Treatment of Rare Diseases

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…

On FA Awareness Day, Friedreich’s Ataxia Research Advocate Is Upbeat

“Excited and beyond belief” is how Ron Bartek, co-founder and president of the Friedreich’s Ataxia Research Alliance (FARA), describes current prospects for finding treatments for the incurable genetic disease that claimed his son, Keith, at age 24. Bartek spoke to Friedreich’s Ataxia News on the sidelines of the recent…

Nonprofit Group Works to Raise Rare Disease Awareness in India

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

Vienna to Host RARE2019 Meeting on Rare Diseases

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

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…

NIH Agency Pioneers Collaborative Research into Rare Diseases

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…

FDA Confirms mFARS as Chief Measure in Phase 2 Trial of Omaveloxolone as Potential FA Therapy

The U.S. Food and Drug Administration (FDA) agreed that Reata Pharmaceuticals can use the modified Friedreich’s Ataxia Rating Scale (mFARS) as a primary endpoint for the second part of its possibly pivotal Phase 2 clinical trial assessing omaveloxolone in treating Friedreich’s ataxia, the company announced. This decision followed Reata’s request that the FDA confirm its earlier guidance that,…