News

Smithsonian Magazine4h
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 ...
BioSpace8h
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 ...
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 ...
A Gene-Editing Breakthrough Saves Infant With Rare Disease
An infant in Philadelphia has been successfully treated with a customized Crispr gene-editing therapy for a rare and deadly ...
HealthShots on MSN15h
2025 ACMG Recap: BioLogic Pharma Solutions Presents Discovery on Rare Disease Patient Identification Using Advanced Genetic Analytics
Genetic specialists from across the globe gathered in the halls of the Los Angeles Convention Center last month for the 2025 ...
BioSpace1d
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 ...
Doctors Achieve Medical Breakthrough Using Gene-Editing Therapy to Heal Baby with Rare Disorder
Doctors have successfully used gene-editing therapy to treat an infant born with a rare disorder. Experts believe the ...
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 ...
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 ...
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 ...
'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 ...
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 ...