The Most Important New Drug of 2012

Cystic Fibrosis Foundation

The Food and Drug Administration looks set for a great 2012.  With a few days left to go, it has approved 40 new drugs and vaccines, one of the most impressive totals ever, according to data from Pharmaceutical Approvals Monthly and FDA press releases. In […]

Noah Faces Fight For Life Against Recurrent Infections Due To Interleukin-12 Deficiency

Noah was born with Interleukin 12 (IL-12) Deficiency and takes interferon therapy

Our Noah Bear: A lot of fight in a tiny body Since our son Noah’s birth, he has continuously been sick with different viral infections and suffered many ear infections resulting in several rounds of antibiotics. In January 2012, Noah had his tonsils and adenoids […]

Children’s Hospital Oakland Receives FDA Clearance to Begin World’s First Cyclodextrin Administration Into the Brains of Twins with Rare and Deadly Cholesterol Disease

Sugar Molecule Used in Common Food and Household Products Like Febreze® Fabric Refresher Called Hydroxypropyl Beta Cyclodextrin (HPßCD) Will be Delivered into Twins’ Central Nervous System in an Attempt to Stop Neurological Progression of Niemann Pick Type C Disease September 23, 2010 Oakland, Calif. – […]

Improve FDA’s Rare Disease Review Process

The Jewish Daily – FORWARD Opinion By Jonathan Jacoby The Jewish community has long been a leader in supporting medical research and education efforts, especially with regard to those diseases that disproportionately afflict people of Ashkenazi Jewish descent. Creating coalitions with other patient advocates in […]

Collaboration is key in rare disease research!

KTVU-TV 2, the leading evening news in the San Francisco Bay Area, today reported on Addi and Cassi Hempel, identical twins affected by a rare disease called Niemann-Pick Type C (NPC). NPC is fatal and degenerative cholesterol disease that is often called the “childhood Alzheimer’s.”  […]