Shooting for the moon: From diagnosis to custom drug, in one year
One weekend in January 2017, Timothy Yu, MD, PhD, was relaxing at home when his wife said, “Hey Tim, will you look at this post on Facebook? This might be something you’d be interested in.” Doctors had just diagnosed a young girl named Mila in Colorado with Batten disease, a rare and fatal neurodegenerative disorder. ... Read More
Opioid alternative? Taming tetrodotoxin for precise painkilling
Opioids remain a mainstay of treatment for chronic and surgical pain, despite their side effects and risk for addiction and overdose. While conventional local anesthetics block pain very effectively, they wear off quickly and can affect the heart and brain. Now, a study in rats offers up a possible alternative, involving an otherwise lethal pufferfish ... Read More
Reviving fetal hemoglobin in sickle cell disease: First patient is symptom-free
Manny Johnson of Boston, 21, previously required monthly blood transfusions to keep his severe sickle cell disease under control. After receiving a new gene therapy treatment, he’s been symptom-free for six months. Researchers at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported Manny’s case Saturday at the American Society of Hematology meeting in San ... Read More