Patients & Caregivers
Advocacy Spotlight: SOLACE organization for LAL Deficiency
The SOLACE organization is a nonprofit group dedicated to increasing shared knowledge about lysosomal acid lipase deficiency (LAL-D).
Advocacy Spotlight: Parent Project Muscular Dystrophy
The mission of Parent Project Muscular Dystrophy (PPMD) is a straightforward one: curing Duchenne muscular dystrophy.
FDA and Medical Devices
Development of Devices for Rare Diseases Remains Challenging
What is Your Definition of Strong?
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.
Newly Approved Kanuma for LAL Deficiency — Interview with Dr Barbara Burton
Kanuma's approval was based partly on a clinical trial led by Barbara Burton, MD, Northwestern University Feinberg School of Medicine in Chicago, Illinois.
Phase 3 Study Testing Volanesorsen in Familial Partial Lipodystrophy Begins
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 Shows Clinical Trial Data for HCM Treatment
MyoKardia, Inc., announced initial clinical data from two Phase 1 trials of MYK-461, which targets the underlying cause of hypertrophic cardiomyopathy (HCM).
PDUFA Date for Alexion's LAL Deficiency Drug Delayed 3 Months
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.
Diffuse Interstitial Myocardial Fibrosis Common in AlstrÃ¶m Syndrome
Results of a recent prospective cohort observational study have revealed that diffuse interstitial myocardial fibrosis is common in AlstrÃ¶m syndrome (ALMS), though not universal.
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