Tag: Wall Street Journal

A total of 10 posts are filed under Wall Street Journal
WSJ Reports: For a Rare Disease, Drug Trials Scramble for Patients
Story by Amy Dockser Marcus After years of effort, scientists and families of young patients with the genetic condition Niemann-Pick Type C… Continue Reading
Brad Margus of Boca Raton, Fla., standing, started a company he hopes will eventually find treatments for the rare neurological disease affecting his sons Quinn, 24, at left, and Jarrett, 25. JOSH RICHIE FOR THE WALL STREET JOURNAL
WSJ Reports: When Parents Start Companies to Cure Their Children
For years, Brad Margus has juggled two goals as chief executive: Make money, and find cures for his children. He just co-founded a startup,… Continue Reading
How Lobbying for Rare Disease Research Influences Congress and NIH
By ED SILVERMAN For years, patient advocates and families have lobbied Congress for more funds to combat rare diseases. But to what extent… Continue Reading
“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… Continue Reading
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… Continue Reading
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… Continue Reading
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… Continue Reading