Rare Disease Report
Physicians
Physicians
Patients & Caregivers

Cardiovascular

In February 2015, the US Food and Drug Administration (FDA) granted orphan drug designation to andexanet alfa, a novel antidote to drugs taken to inhibit factor Xa (FXa).
In February 2015, the US Food and Drug Administration (FDA) granted orphan drug designation to andexanet alfa, a novel antidote to drugs taken to inhibit factor Xa (FXa).
Ehlers-Danlos syndrome (EDS) is a heterogeneous group of inherited connective tissue disorders that can vary from loose joints to life-threatening complications, depending on type. The vascular form of EDS, which is estimated to affect 1 in 250,000 people
Candace Moose, Co-Founder and President of the Board of the Myocarditis Foundation talks about the origins of the foundation and its current offerings to help advance awareness and research for myocarditis.
Lysosomal acid lipase (LAL) deficiency is an autosomal recessive lysosomal disease caused by decreased activity of the LAL enzyme.
The US Food and Drug Administration (FDA) granted Abiomed's application for a humanitarian device exemption for the Impella RP System, a percutaneous medical device designed to assist the heart with circulation in adult or pediatric patients who have right heart failure or inadequate blood flow from the right side of the heart.
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.
Homozygous familial hypercholesterolemia (HoFH) is an exceedingly rare, life-threatening autosomal dominant disorder characterized by markedly elevated low-density lipoprotein cholesterol (LDL) levels (>500 mg/dL).
Copyright © RareDR 2013-2017 Rare Disease Communications. All Rights Reserved.