President of the Progeria Research Foundation (PRF), Audrey Gordon, discusses the clinical trials PRF is involved with, as well as the role PRF is playing in getting as many progeria patients as possible on a treatment.
Aegerion Pharmaceuticals has acquired metreleptin (Myalept), a drug approved in February 2014 as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
On the heels of positive findings from a randomized, double-blind, phase III trial comparing sebelipase alfa (SBC-102) with placebo in individuals with lysosomal acid lipase (LAL) deficiency, Synageva BioPharma submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) in December 2014 with a request for priority review.
Gordon and Gordon's editorial "The Progeria Research Foundation: Its Remarkable Journey From Obscurity to Treatment"is a must read for the rare disease community. And the key takeaway message was quite simple...Working together is exponentially better than working alone.