Therapy May Curb Kidney Deterioration in Patients With Rare Disorder, Methylmalonic Acidemia


July 29, 2013 — A team led by researchers at the National Institutes of Health has overcome a major biological hurdle in an effort to find improved treatments for patients with a rare disease called methylmalonic acidemia (MMA). Using genetically engineered mice created for their studies, […]

Forbes: The Lifesaving Promise of the Pharmaceutical Drug Pipeline

(Photo credit: Images_of_Money)

There was a time not long ago when patients suffering from rare diseases had little hope of finding a medicine that could cure their ills. But in the 30 years since Congress passed the Orphan Drug Act, research into “orphan drugs” — defined as those […]

The Myelin Repair Foundation Aims to Eliminate The Valley of Death


What a great video. Watch to learn why $139 billion dollars invested into medical research only leads to 21 FDA approved drugs. The Myelin Repair Foundation is one of many organizations/RARE advocates working to change this. To learn more about the Myelin Repair Foundation, click […]

AKU Society joins forces with Partnership for Cures to find AKU treatments

The AKU Society has joined forces with US charity Partnership for Cures in order to develop and fund clinical trials into treatments for AKU. Partnership for Cures drives the re-use of existing drugs for untreated conditions through a collaboration of philanthropists, disease specific charities, patient […]

Tailoring Treatments for Kids; Perspectives Podcast Series a discussion with Don Lombardi

In treating pediatric patients, doctors and hospitals are often limited by available devices or drugs that have not been optimized for the treatment of children. Don Lombardi saw the absence of appropriate devices and drug formulations for kids as a significant opportunity to effect positive […]