CAMP4 Therapeutics Prepares CMP-002 Phase 1/2 Trial for SYNGAP1
WATERTOWN, Mass. — September 8, 2026 CAMP4 Therapeutics Corporation announced plans to host a virtual Analyst Day on September...
rare disease
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BridgeBio Advances Oral Infigratinib Toward Achondroplasia Launch
PALO ALTO, Calif. — September 9, 2026 BridgeBio Pharma, Inc. presented new exploratory analyses from the Phase 3 PROPEL...
Orphalan Launches Phase 3 Trial of Once-Daily Trientine
Chicago, Illinois, USA, September 8, 2026 Orphalan has initiated the global Phase 3 TRADITiONAL clinical trial evaluating an investigational...
Intellia Therapeutics Gains FDA Priority Review for Lonvo-z in HAE
CAMBRIDGE, Mass. — September 8, 2026 Intellia Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted...
Pharvaris Reports Positive Phase 3 CHAPTER-3 Data for Deucrictibant XR
ZUG, Switzerland — September 8, 2026 Pharvaris announced positive topline results from the CHAPTER-3 pivotal Phase 3 study evaluating...
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Belite Bio Files Japan NDA for Tinlarebant in Stargardt Disease
San Diego, California, U.S., September 8, 2026 Belite Bio has announced the submission of a New Drug Application (NDA)...
Alkeus Reports Positive Gildeuretinol Results in Stargardt Disease
Cambridge, Massachusetts — September 3, 2026 Alkeus Pharmaceuticals announced the publication of TEASE-1 efficacy and safety results in JAMA...
uniQure Submits BLA for AMT-130 in Huntington’s Disease
LEXINGTON, Mass. and AMSTERDAM, September 2, 2026 uniQure N.V. announced the submission of a Biologics License Application (BLA) to...
AstraZeneca Tagrisso Combo Improves Lung Cancer PFS
Cambridge, UK, September 1, 2026 AstraZeneca has announced positive high-level results from the Phase III SANOVO trial, showing that...
Ascendis, BioMarin Reach Global YUVIWEL License Deal
COPENHAGEN, Denmark, August 31, 2026 Ascendis Pharma A/S and BioMarin Pharmaceutical Inc. have entered into a binding term sheet...
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