FDA questions the efficacy of Capricor’s Duchenne drug
The Food and Drug Administration said Monday that Capricor Therapeutics’ stem cell treatment for Duchenne muscular dystrophy did…
Rare disease linked to rabbits has Colorado health officials on alert
A rare but potentially serious disease tied to rabbits and ticks has been detected in northern Colorado, prompting…
GOP Sen. Johnson investigating FDA rare disease drug rejections
WASHINGTON — Sen. Ron Johnson (R-Wis.) said he is investigating the Food and Drug Administration’s rejections of rare disease…
Boy, 2, Dies Suddenly from Rare Disease with Flu-Like Symptoms, Weeks Before His Birthday: He ‘Had the Biggest Personality’
NEED TO KNOW A 2-year-old boy died from a rare disease with flu-like symptoms, just weeks before his…
Gene therapy trial death unnerves promising area of brain research
Gene therapy researchers were converging on a holy grail. A few years ago, researchers at labs and companies…
A Rogue Gene Explains Mysterious Cases of Diabetes and Epilepsy in Newborns
Credit: Wikimedia Commons When six infants around the world were diagnosed with an odd trio of symptoms (diabetes,…
Rare genetic mutation that brings threat of brain bleeding unusually common in New Mexico | Local News
For Sakura Tafoya, it started with a header. The Santa Fe girl and her family were playing soccer…
Baylor Genetics Presents New Data on Clinical and
HOUSTON, Oct. 15, 2025 (GLOBE NEWSWIRE) — Baylor Genetics, a clinical diagnostic laboratory at the forefront of genetic…
Oxford Nanopore Announces PromethION Plus Flow Cell and Human Genetics Updates at ASHG 2025
OXFORD, England, October 15, 2025–(BUSINESS WIRE)–At the American Society of Human Genetics (ASHG) 2025 industry session on 16th…
Baylor Genetics Presents New Data on Clinical and Diagnostic Utility of RNA Sequencing for Rare Disease at the American Society of Human Genetics 2025 Annual Meeting
Baylor Genetics Research underscores clinical utility of RNA sequencing in providing critical insights to drive diagnoses and medical…
FDA approves “lifesaving” drug after Omaha mom’s pleas
An Omaha mom is celebrating the Food and Drug Administration’s approval of elamipretide for Barth syndrome.The sign Jordan…
Medicines Reimagined – Repurposing Drugs For New Indications
In a group of health-related headlines, one says “Hope for new drug approach. getty As government-supported research in…