News

A Gene-Editing Breakthrough Saves Infant With Rare Disease
The Crispr therapy was tailored to the baby’s specific condition and could provide a template for others.
Child with rare disease will still get vital drug
Nine-year-old Addy Clarke, from Swindon, has Batten Disease - a rare degenerative disease that has no cure. An agreement has ...
BioSpace13h
CRISPR Success in Single Baby Highlights Rare Disease ‘Crisis’
Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to ...
Smithsonian Magazine on MSN10h
In a Remarkable First, a Baby With a Rare Disease Receives Personalized Gene Therapy
Researchers say the CRISPR-based technique used could eventually be employed to treat more people with rare genetic diseases ...
BioSpace2d
Makary’s ‘Conditional Approval’ Pathway for Rare Diseases Poses More Questions Than Answers
While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner ...
Fierce Biotech1d
Applied's rare disease drug suffers another phase 3 failure months after FDA rejection
Applied Therapeutics’ rare disease drug has failed another late-stage clinical trial, adding to the woes of a candidate that ...
San Francisco boy with rare bone marrow disease still searching for donor
A San Francisco boy with a rare bone marrow disease has been on the waitlist for more than a year. Gia Vang reports. A San ...
Managed Healthcare Executive1d
Breakthrough Gene Therapy Saves Infant with Rare Metabolic Disease
A personalized gene editing therapy was successfully used to treat an infant with CPS1 deficiency, a disease that causes ...
Healio4d
Infant with rare disease receives world’s first personalized gene-editing therapy
An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...
Gene editing cures child of rare disease in world first
Family overjoyed correction to DNA defect that causes metabolic disorder has given KJ Muldoon a new lease of life ...
Rare Disease Patients Await Congressional Action on Life-Saving Treatments
President Trump recently signed a wide-ranging executive order urging Congress to amend a crucial provision of a Biden-era ...
'Everybody encouraged me to drop out': Omaha woman perseveres through rare illness, getting her degree
When an Omaha woman was diagnosed with a rare illness as a teenager, it affected not only her health but also her high school ...