Posts by: Larry Luxner

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated ... Read more

March 1, 2018March 1, 2018

Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children’s National

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in ... Read more

February 27, 2018

BioNews to Cover 3 Rare Disease Day Events, Including NIH Conference

In recognition of Rare Disease Day 2018, BioNews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies ... Read more

February 22, 2018

Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and ... Read more

February 13, 2018March 15, 2018

Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress

David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic ... Read more

January 30, 2018January 30, 2018

NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least ... Read more

November 21, 2017November 21, 2017

Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as ... Read more

November 2, 2017

Children’s National, NORD Partner to Create Rare Disease ‘Centers of Excellence’

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the ... Read more

October 23, 2017October 23, 2017

#NORDsummit – Alternatives to Large Placebo Trials, Grant Awards Among Ways FDA Supporting Rare Diseases, Chief Says

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in ... Read more

October 19, 2017

#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies ... Read more