CLINICAL ROLE -
Famed Writer Dying of Liver Cancer
Writer and neurologist Oliver Sacks confirms he has terminal cancer of the liver.
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FDA Proposes Changes to Expanded Access Procedure
Changes address concerns that the process to access investigational drugs was too difficult.
Lundbeck's Northera Now Available Through Specialty Pharmacy
New treatment option for treatment of orthostatic dizziness.
FDA Approves Pfizer's Gaucher Drug for Children
Elelyso offers pediatric patients with Type 1 Gaucher disease another treatment option.
Hemophilia A Phase 3 Trial Update
Treatment reduces annualized bleed rates in the treatment arm compared to the on-demand treatment arm.
CureDuchenne Gives Prosensa $7 Million To Restart Trials
Philanthropic efforts could accelerate drug development and treatment.
Eloctate Approved for Hemophilia A
Drug seeks to mitigate challenge of infusion frequency while protecting against bleeding episodes.
Sarepta Provides Update on Clinical Trials Planned for 2014
Streamlining exon skipping drugs for other mutations will allow the company to reach over half of the Duchenne muscular dystrophy population.
Farnesylation Inhibitors Allow Children with Progeria To Live Longer
Study finds progeria treatment to extend survival time by 1.6 years.