Rare Disease Report
Patients & Caregivers


The SOLACE organization is a nonprofit group dedicated to increasing shared knowledge about lysosomal acid lipase deficiency (LAL-D).
The mission of Parent Project Muscular Dystrophy (PPMD) is a straightforward one: curing Duchenne muscular dystrophy.
Development of Devices for Rare Diseases Remains Challenging
A photo of 29 year old professional powerlifter David "The Beast" Douglas, and 12 year old Progeria patient, Lindsay Ratcliffe has gone viral.
Although the vast majority of cardiological conditions are fairly common or very common, rare cardiovascular problems continue to strike people around the globe.
Kanuma's approval was based partly on a clinical trial led by Barbara Burton, MD, Northwestern University Feinberg School of Medicine in Chicago, Illinois.
Familial partial lipodystrophy is a rare, genetic disorder in which patients are unable to store fat or triglycerides in normal fat stores so excess triglycerides builds up in various organs and the blood.
MyoKardia, Inc., announced initial clinical data from two Phase 1 trials of MYK-461, which targets the underlying cause of hypertrophic cardiomyopathy (HCM).
The FDA extended the PDUFA date to allow additional time to review the new CMC information. The FDA has not asked for additional clinical data.
Results of a recent prospective cohort observational study have revealed that diffuse interstitial myocardial fibrosis is common in Alström syndrome (ALMS), though not universal.
Copyright © RareDR 2013-2017 Rare Disease Communications. All Rights Reserved.