We have incredible news to share! Massachusetts-based bluebird bio, a leader in the research and treatment of cancer and severe genetic diseases, just received FDA “Breakthrough Therapy” designation for their Lenti-D™ gene therapy for patients with cerebral adrenoleukodystrophy (CALD). This designation will allow for expedited development and review of the treatment protocol, meaning it is …Read More
Jean and Brian recently traveled to Baltimore for the exciting inauguration of The Moser Center at The Kennedy Krieger Institute, an internationally recognized institution dedicated to improving the lives of individuals with disorders of the brain, spinal cord, and musculoskeletal system. The Moser Center for Leukodystrophies is named in honor of the late Dr. Hugo …Read More
We had a great day with Mrs. Savoia and Mrs. Schingh’s fifth graders at Green Acres School in North Haven, discussing the passage of our bill and the importance of newborn screening, and the addition of ALD to the existing newborn screening panel.
Sad news today – Augusto Odone, the World Bank economist who defied the medical establishment to treat his son Lorenzo with ’Lorenzo’s Oil’ has passed away. Odone gained recognition with his wife Michaela for their efforts to keep Lorenzo alive after they learned of his ALD diagnosis and its devastating progression. The Odones’ story was …Read More