News

Doctors save baby's life with first-ever gene fix for deadly rare disease
The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific ...
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 ...
BioSpace10h
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 ...
EXCLUSIVE: Telomir Pharma To Raise $3 Million Via Equity Financing To Fund Its Rare Disease IND
Telomir Pharma (NASDAQ: TELO) secured $3M in equity financing from Bayshore Trust, its largest shareholder, at $3 per share ...
Telomir Pharmaceuticals Secures $3 Million at a Premium in Straight Equity Sale Involving No Warrants to Advance Rare Disease IND
Investment from largest shareholder strengthens balance sheet, signals insider conviction, and funds Telomir-1’s upcoming IND submission for a rare disease indication ...
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 ...
Smithsonian Magazine on MSN1d
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 ...
Fierce Biotech2d
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 ...
'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 Executive2d
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 ...
Healio5d
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 ...