Browsing Tag
Rare diseases
36 posts
Can VGA039 become a once-monthly standard of care for von Willebrand disease?
Star Therapeutics' VGA039 shows strong bleeding reduction across all VWD types in Phase 1/2 trial. Discover how this monthly shot could change the standard of care.
AstraZeneca (LSE: AZN) goes big on U.S. supply chain with latest $2bn manufacturing push
AstraZeneca invests $2B in Maryland to expand biologics and rare disease drug manufacturing. Find out what this means for U.S. jobs and the pharma supply chain.
Atossa Therapeutics (NASDAQ: ATOS) expands (Z)-endoxifen to DMD: Will investors buy the thesis?
Atossa Therapeutics eyes new ground in DMD care with (Z)-endoxifen. Find out how this small molecule could reshape treatment for carriers and boys alike.
Quoin Pharmaceuticals (NASDAQ: QNRX) spikes over 50% after rapamycin breakthrough in rare disease program
Quoin Pharmaceuticals (NASDAQ: QNRX) stock surged over 50% after achieving key rapamycin delivery targets. Find out how this positions its rare disease pipeline.
Novartis makes $12bn bet on RNA medicine with Avidity Biosciences acquisition to expand rare-disease pipeline
Find out why Novartis’s $12 billion Avidity Biosciences deal marks a turning point for big pharma’s RNA-therapy ambitions.
Neuren Pharmaceuticals secures third FDA Fast Track for NNZ-2591 as shares rise 4.21%
Find out how Neuren Pharmaceuticals is expanding FDA Fast Track coverage for NNZ‑2591 across rare disorders like PMS, AS, and Pitt Hopkins.
What is Niemann–Pick disease and why is this rare genetic disorder so hard to treat?
Discover Niemann–Pick disease, a rare genetic disorder affecting lipid metabolism. Learn about its types, symptoms, causes, diagnosis, and latest treatments.
Why Avanzanite’s hire of Dr. Mark Bechter could reshape how orphan drugs reach patients across 32 countries
Avanzanite Bioscience hires Dr. Mark Bechter as SVP medical affairs to drive rare disease growth, with H1 2025 revenues tripling. Learn more.
Vonvendi gets green light for pediatric bleeding disorder care in FDA expansion
Find out how the FDA's expanded approval of Vonvendi opens new treatment avenues for children and adults with all types of von Willebrand disease.
Theravance completes enrollment in Phase 3 CYPRESS trial for ampreloxetine in MSA-related nOH
Theravance completes Phase 3 enrollment for ampreloxetine in MSA-related nOH. Topline data expected in Q1 2026. Could this be a rare disease breakthrough?