A Glimmer of Hope: Groundbreaking Gene Therapy Dramatically Reduces Huntington's Protein

A Beacon of Hope: uniQure's AMT-130 Gene Therapy Shines in Huntington's Disease Trial

Elara's Journey: A Community Rallies Behind an Alberta Toddler's Hope for a Cure in Italy

MBX Biosciences Rockets Skyward: Breakthrough Hypoparathyroidism Drug Unlocks Massive Pre-Market Surge!

MBX Biosciences Unveils Groundbreaking Promise for Hypoparathyroidism Patients with Novel Experimental Drug

Palvella Therapeutics' QTORIN: A Glimmer of Hope Nears for Pachyonychia Congenita Sufferers

A New Horizon for Duchenne: FDA Approves First Gene Therapy for Life-Threatening Disease in Boys

KalVista's Game-Changing HAE Drug Gets Green Light in EU & Switzerland, Stock Set to Explode!

Kerala's Brave Fight: Four Defy the 'Brain-Eating Amoeba' in Kozhikode

Kerala Mourns as Rare Brain-Eating Amoeba Claims Another Life: Understanding the Threat of PAM

Game-Changer Alert: Travere Therapeutics Shares SOAR as FDA Fast-Tracks Rare Kidney Disease Drug Sparsentan!

From Gaza's Shadow to Italy's Embrace: A Toddler's Miraculous Recovery from Rare Disease and Malnutrition

Kerala Teen's Miraculous Escape: Surviving the World's First Dual Amoebic and Fungal Brain Infection

The Unyielding Quest: NJ Parents Defy Big Pharma to Forge Cures for Their Children

A Silent Predator's Toll: Missouri Patient Succumbs to Brain-Eating Amoeba

Unraveling Bloom Syndrome: A Comprehensive Guide to a Rare Genetic Condition

Rare Plague Case Confirmed: California Resident Recovers Amidst Public Health Scrutiny

A New Hope for Danon Disease: FDA Lifts Clinical Hold on Rocket Pharma's Groundbreaking Gene Therapy

A New Dawn for Rare Disease Treatment: FDA Approves Groundbreaking Gene Therapy for DEB