ACTION ALERT – World RARE Disease Day at National Institute of Health
Rare Disease Day at NIH (RDD@NIH) On February 29, 2012, the National Institutes of Health (NIH) will celebrate the fifth annual Rare Disease Day with a day-long celebration and recognition of the various rare diseases research activities supported by the NIH Office of Rare Diseases Research, the NIH Clinical Center, other NIH Institutes and Centers; [...]
Wall Street Journal Reports – Do It Yourself Drug Development, With Some Help!
The effort by two families to buy and develop a drug that holds promise in treating Duchenne muscular dystrophy — described today in the WSJ — is the result of an innovative new model set up to support the burgeoning phenomenon of do-it-yourself drug development. Before the Seckler and Wicka families bought halofuginone, a drug that [...]
Guest Blog Scientific American – Innovation in Medical Diagnositics Saves Lives
Innovation in Medical Diagnostics Saves Lives – a system that isn’t broken and doesn’t need fixing For over a century, the U.S. Food and Drug Administration (FDA) has done a laudable job of ensuring the safety and efficacy of healthcare for Americans. Recently announced potential policy changes, however, have a good chance of doing just [...]
'THE HILL' Reports: Help children with rare diseases get the new medicines they need
Having a seriously ill child is always difficult and painful. It’s even more so when a child’s illness is one of the 7,000 rare diseases for which cures and treatments are hard to find. Rare diseases affect more than 15 million children in the United States. A disease is considered rare if it affects less [...]
Make Your Voice Heard
Howard is collecting your stories, comments, recommendations, etc. for the FDA’s hearing on rare disease in June 2010. Howard will also be representing MarbleRoad at the NORD Partners in Progress 2010 Gala in Washington DC on May 18, 2010.
Good news from the FDA (yes, the FDA!)
Transformation is happening – FDA and rare disease! We have gotten so accustomed to hearing criticisms of the The U.S. Food and Drug Administration which, like most criticism, is sometimes valid, sometimes not). Here are two new developments worth talking about! First, in an admirable show of cooperation, the FDA reached an agreement with the [...]
CureDuchenne Hopes FDA Will Accomodate New Class Of Muscular Dystrophy Drugs Upon Proof Of Safety
Seven years ago, we were a happy family with mom, dad and a beautiful 5 year old boy named Hawken. We had noticed our son was unable to keep up in his first soccer team but we were assured by his pediatrician he was just a “late bloomer.” After insisting on some testing, we got [...]