News

Minovia Therapeutics Announces FDA Fast Track and Rare Pediatric Disease Designations for MNV-201 in Pearson Syndrome
Regulatory Milestone Underscores Urgency and Promise of First-in-Class Mitochondrial Cell TherapyHaifa, ISRAEL, June 30, 2025 (GLOBE NEWSWIRE) -- Minovia Therapeutics Ltd. (“Minovia” or the “Company”) ...
Minovia Gets FDA Designations Supporting Development of Pearson Syndrome Treatment
Minovia Therapeutics said Monday that the Food and Drug Administration granted fast-track and rare-pediatric-disease designations to its lead investigational compound. The drug, MNV-201, is in Phase 2 ...
The Manila Times4d
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has ...
TMCnet2d
Belite Bio Announces Completion of Enrollment in the Pivotal Global Phase 3 PHOENIX Trial Evaluating Oral Tinlarebant in Geographic Atrophy
The PHOENIX study is a 24-month, randomized, double-masked, placebo-controlled, multicenter, pivotal Phase 3 trial. The study is ongoing across sites in the United States, the United Kingdom, France, ...
Neurofibromatosis Type 2 Market Set For Robust Growth Through 2034, Driven By Innovative Therapies And Growing Disease Awareness Delveinsight
Neurofibromatosis Type 2 Market Insight, Epidemiology And Market Forecast - 2034The Neurofibromatosis Type 2 treatment market is experiencin ...
Community Impact4d
6 hospital system updates for the Northwest Austin area
Here are the latest updates for six hospital systems across the Northwest Austin area. 1. Ascension Seton Northwest Hospital ...
Parliamentarian reverses course on rare disease drug price provision in tax bill
The Senate Parliamentarian reversed course on a small provision in the One Big Beautiful Bill Act that could boost cures for ...
Research Moves Slowly. Rare Diseases Don't—So Patients Aren't Waiting
Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?
The Jewish Press4d
Researchers at Tel Aviv University Develop Groundbreaking Model for Rare Genetic Disease
Prof. Moran Hausman-Kedem, a pediatric neurologist involved in the research, emphasized the clinical value of the model: “For rare diseases where patient populations are too small for large-scale ...
Nearly 150 new Florida laws went into effect July 1. Here's what each one means
Nearly 150 new Florida laws went into effect on July 1 after Gov. Ron DeSantis signed 11 more the day before. Here is what ...
Spectrum News NY123h
Growing tick population draws attention to rare Lyme symptom - NY1
Explosion in the tick pop­u­lation draws attention to rare Lyme disease symptoms in children BY Erin Billups - National Health Reporter Nationwide UPDATED 7:35 PM ET Jun. 21, 2021 ...