News

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 MSN1d
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 ...
Mirage News2h
World First: Prime Editing Reverses Rare Illness
This spring at CHU Sainte-Justine, an 18-year-old patient with a rare disease called chronic granulomatous disease (CGD) became the first person ever to receive next-generation gene therapy known as ...
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 ...
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 ...
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 ...
'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 ...
Scientist verdict on 'rare incident' as mad cow disease found on UK farm
Creutzfeldt-Jakob disease (CJD) is a rare and fatal condition that affects the human brain, causing brain damage that worsens ...
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 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 ...