ANCA Features

At 40, Orphan Drug Act Enjoys Rare Bipartisan Support

Larry Luxner

January 18, 2023

Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.

ALGS Features, ANCA Features, Features, LAL-D Features, LAL-D News Briefs, News Briefs, Pompe Features, WD Features

EveryLife Foundation Navigates Complex Issues to Advocate for Rare Disease Patients

Larry Luxner

November 21, 2022

The EveryLife Foundation for Rare Diseases pushes a legislative agenda aimed at benefitting the 30 million Americans with a rare disease.

ANCA Features, Features, LAL-D Features, MS Features

Synthetic Control Arms Could Facilitate More RCTs in Rare Disease

Tori Rodriguez, MA, LPC, AHC

November 14, 2022

Dr. Rahul Banerjee of the University of Washington discusses the benefits and drawbacks of using synthetic control arms in randomized controlled trials.

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ANCA Features

At 40, Orphan Drug Act Enjoys Rare Bipartisan Support

EveryLife Foundation Navigates Complex Issues to Advocate for Rare Disease Patients

Synthetic Control Arms Could Facilitate More RCTs in Rare Disease

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