BioCryst Pharmaceuticals Strikes Strategic $700 Million Deal, Propelling Astria's HAE Pipeline and Strengthening Its Own Focus

FDA Grants Priority Review to Rocket Pharma's Groundbreaking Gene Therapy for Rare LAD-I

FDA Extends Review for Denali's Hunter Syndrome Breakthrough: A Closer Look at Tividenofusp Alfa's Path to Approval

Kerala on High Alert: Third Brain-Eating Amoeba Death in 11 Days Sends Public Health Officials Scrambling

Unearthing Medieval Ireland's Medical Mysteries: A 12th-Century Skeleton Reveals Ancient Plagues

Monopar's Breakthrough in Wilson Disease: A New Dawn with AstraZeneca's Shadow

Biohaven's Breakthrough Bid: Navigating FDA Flexibility for Spinocerebellar Ataxia's Future

The Shocking Truth Behind Her 'Massage Gun' Bruises: A Rare Diagnosis from the Past

Taysha Gene Therapies' TSHA-102 Receives FDA Breakthrough Status: A New Hope for Rett Syndrome

A Heartbreaking Loss: Sonu Sood Mourns the Passing of Young Abhijot Singh After Valiant Battle

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

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

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

A Beacon of Hope: Toddler Karim's Miraculous Recovery After Gaza Evacuation

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