“A Story Six Years in the Making” Wall Street Journal Produces Masterful Piece on the Hempel Family Saga


With graphics unlike you’ve ever seen before, the Wall Street Journal has created a multi-chapter journey for readers to explore the world of rare disease and treatment through the Hempel family’s journey. The piece, which includes incredible photography, video, interviews, and a complete history of […]

Wall Street Journal Goes In Depth On Tay-Sachs – Rare Disease Does Not Only Afflict Jewish, But Irish Community


Study Looks at Irish Risk for a Rare Fatal Disease By Amy Dockser Marcus The Wall Street Journal Online Tay-Sachs disease is generally known as a Jewish genetic disorder. Now, researchers are beginning to focus on another community at risk: people of Irish descent. Tay-Sachs […]

Change is Needed at the FDA Says Andrew Von Eschenbach


“Breakthrough technologies deserve a breakthrough in the way the FDA evaluates them.”  - Andrew Von Eschenbach Mr. Von Eschenbach couldn’t have stated it any clearer.  On February 14, the Wall Street Journal posted an on-line article by Mr. Eschenbach titled: Medical Innovation: How the U.S. […]

Do It Yourself Drug Development, With Some Help! – Wall Street Journal Reports


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 […]

Do We Need a War on Rare Diseases?

By Amy Dockser Marcus, Wall Street Journal Is it time to declare a national “war on rare diseases” comparable to the famous “war on cancer” that began in the 1970s? The Institute of Medicine report released today stops short of recommending a full martial assault, […]

Increased Patient Participation in the Fight Against Rare Disease – IOM Report


The Institute of Medicine (IOM) issued a new report: “Rare Diseases and Orphan Products: Accelerating Research and Development” which calls for increased participarticipation by people with rare diseases.

Bugged by Genzyme?

The big buzz in the Rare Disease community over the past few days has been Genzyme’s announcement to halt production and shipments of Cerezyme and Fabrazyme, two drugs for rare genetic disorders Gaucher disease and Fabry disease, after a virus (Vesivirus 2117) was discovered in production equipment at its […]