Measuring the Value of Therapies for Ultra-Rare Diseases

  The pricing of drugs is characterized by a…

Incredible Teen with Ataxia Telangiectasia Raising Money for A-T With Quilt Project
CATHRYN’S QUILT Cathryn Achilles (age 19) was diagnosed at age 8 with ataxia telangiectasia or A-T. She loves to make quilts and although… Continue Reading
A NEW START FOR GENE THERAPY FOR 'BUBBLE BOY' DISEASE Children's Hospital Boston Reports
First U.S.-treated patient doing well! Agustín has a form of X-linked Severe Combined Immunodeficiency (SCID-X1), better known as “bubble… Continue Reading
Batten Disease Awareness Weekend Bringing Hope for Children with a Fatal Disease—and No Cure in Sight
June 1, 2011 (Reynoldsburg, OH) – Batten Disease Support and Research Association (BDSRA) is holding its 3rd annual Batten Disease… Continue Reading
Certain Proof: A Question of Worth, Debuts Saturday June 4 in Hollywood
Director Ray Ellis will join friends and movie go-ers this Saturday at a special premiere of his movie “Certain Proof: A Question of… Continue Reading
NEW OPPORTUNITIES FOR ANGELMAN SYNDROME – Children's Hospital Boston Reports
Angelman Syndrome is an rare, neurogenetic condition that falls into the category of orphan diseases. Children’s Hospital Boston is… Continue Reading
GLOBAL GENES PROJECT HOLDS INAUGURAL BOARD MEETING
Participants from all over the country converge on Southern California to lay the groundwork for the Global Genes Project’s continued… Continue Reading
NEW DOCUMENTARY: RARE DISEASE THE FOCUS – BE INFORMED AND INSPIRED
Swell is a documentary film that showcases the lives of people suffering from a rare, genetic disorder, Hereditary Angioedema (HAE). Upon… Continue Reading