Tagged as: Lorenzo’s Oil

bluebird bio to Present Clinical Data from Study of Lenti-D in ALD at CNS Annual Meeting

CAMBRIDGE, Mass.–(BUSINESS WIRE)–bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, today announced that interim data from the initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating Lenti-D™ investigational

Read More

Forbes Health Writer, Rita Rubin, on ‪Lorenzo’s Oil‬, the Odones, & ‪‎ALD‬ ‪Newborn Screening

Lorenzo’s Oil Could Not Cure Lorenzo, But Newborn Screening Is Expected To Save Others From His Fate By: Rita Rubin Forbes.com Lorenzo’s Oil, a 1992 film starring Nick Nolte and Susan Sarandon, depicted Augusto and Michaela Odone’s quest for a treatment that could save their son Lorenzo from dying of

Read More

The Myelin Project & Brian’s Hope Grant $50,000 to Dr. Raymond’s Lab

In partnership with Brian’s Hope Foundation, The Myelin Project granted $50,000 to Dr. Gerald Raymond‘s lab at the University of Minnesota. Raymond’ lab works in collaboration with the Inherited Metabolic and Storage Disease Bone Marrow Transplantation Program, the Center for Orphan Drug Research, the Center for Magnetic Resonance Research, and others,

Read More