James Radke, Rare Disease Report

Writer and neurologist Oliver Sacks confirms he has terminal cancer of the liver.

Changes address concerns that the process to access investigational drugs was too difficult.

New treatment option for treatment of orthostatic dizziness.

Elelyso offers pediatric patients with Type 1 Gaucher disease another treatment option.

Treatment reduces annualized bleed rates in the treatment arm compared to the on-demand treatment arm.

Philanthropic efforts could accelerate drug development and treatment.

Drug seeks to mitigate challenge of infusion frequency while protecting against bleeding episodes.

Streamlining exon skipping drugs for other mutations will allow the company to reach over half of the Duchenne muscular dystrophy population.

Study finds progeria treatment to extend survival time by 1.6 years.