Familial Hypercholesterolemia
Access Health to Air New Segment on Familial Hypercholesterolemia on 3/29

Don’t miss tomorrow’s segment on Access…

CNN Story Highlights Global Problem Facing Millions Suffering From Rare Diseases
CNN is running a comprehensive package about rare diseases and the crisis patients with rare diseases are facing with by being priced out… Continue Reading
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… Continue Reading
Re-Thinking Drug Development – NIH Announces New Institute and Pledges Millions To Develop Therapies for Rare Diseases
With the cost of bringing a new drug to market now exceeding $1 billion dollars and taking up to 15 years to test and develop, global… Continue Reading
The Myelin Project Announces $300,000 Challenge Grant To Spur New Research Into Adrenomyeloneuropathy (AMN)
Augusto and Michaela Odone founded The Myelin Project in 1989. Their son, Lorenzo of the movie “Lorenzo’s Oil,” was diagnosed at the… Continue Reading
NORD Partners in Progress Rare Disease Summit – Update
NORD hosted a rare disease summit in Washington DC with the goal of setting a policy agenda to help create a more responsive environment for… Continue Reading
A Fine Line Between Research and Parenting – Living With Neuronopathic Gaucher's Disease
Not only does my beautiful little girl Hannah have an incredibly rare and fatal disease of which only a few hundred children in the world… Continue Reading
A Mom Brokers Treatment For Her Twins’ Rare and Fatal Illness
Bucking Scientific Convention, Ms. Hempel Gets Researchers From Different Fields to Share Data on Potential Therapy By AMY DOCKSER MARCUS… Continue Reading