General Information On Clinical Trials

Orphan Drug Designation A drug becomes an “orphan” when it receives orphan designation from the Office of Orphan Products Development, FDA. An orphan drug is a drug intended to treat diseases or conditions affecting fewer than 200,000 people per year in the United...

Phase II Pilot Trials Begin (January 1999) in the Netherlands

Following is background information and a Q & A released by the Pharming/Genzyme LLC on important issues concerning clinical trials for enzyme replacement therapy now underway in the Netherlands on acid maltase deficiency, Pompe’s disease. Background—Pompe Disease...