News

BioSpace9h
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 ...
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 ...
Smithsonian Magazine on MSN2d
In a Remarkable First, a Baby With a Rare Disease Receives Personalized Gene Therapy
For the first time, doctors have treated an infant with a rare genetic disease with a gene-editing therapy tailored ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
Alan Alda Shares Update on Life With Parkinson’s Disease and Prosopagnosia: ‘Making Progress’
Alan Alda shared a rare health update about his battle with Parkinson’s disease 10 years after he was first diagnosed. In an ...
What Is Alan Jackson's Health Condition? Inside the Country Star's Decade-Plus Journey with a Rare Disease
Alan Jackson was diagnosed with Charcot-Marie-Tooth disease, a degenerative nerve condition, in 2021. Here's everything to ...
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 ...