Italfarmaco Secures Managed Entry Agreement for Duvyzat in Georgia
MILAN, Italy, July 31, 2026 talfarmaco S.p.A. announced a managed entry agreement with the Georgian Ministry of Health to...
rare disease
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Saol Therapeutics Advances SL1009 in FDA Review
ROSWELL, Ga., DUBLIN, Ireland, and HAMILTON, Bermuda, July 28, 2026 Saol Therapeutics has announced that the U.S. Food and...
United Therapeutics Reports Phase 3 Ralinepag Results in The Lancet
SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C., July 29, 2026 United Therapeutics Corporation announced that the complete results...
Dasher Neuroscience Advances YA-101 Phase 2 Trial
TAIPEI, July 28, 2026 Dasher Neuroscience Holdings Inc. has successfully completed patient enrollment in its global Phase 2 clinical...
Medicus Pharma Receives FDA Clearance for Registrational SkinJect Study
PHILADELPHIA, July 27, 2026 Medicus Pharma Ltd. announced that the U.S. Food and Drug Administration (FDA) has issued a...
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BioMarin Partners n-Lorem on First ReNU Syndrome Therapy
SAN RAFAEL, Calif. & SAN DIEGO, July 27, 2026 BioMarin Pharmaceutical Inc. and the n-Lorem Foundation have announced a...
Alexion, AstraZeneca Rare Disease Shares Phase 3 Ultomiris Trial Results
BOSTON, Mass., July 27, 2026 Alexion, AstraZeneca Rare Disease announced high-level results from its global Phase 3 clinical program...
AnHorn Medicines Advances AH-008 into Phase I Trial for CIPN Prevention
Irvine, California, USA — July 27, 2026 July 27, 2026 – AnHorn Medicines has announced the successful first subject...
Zevra Therapeutics to Seek EU Re-Examination for Arimoclomol After CHMP Opinion
BOSTON – July 24, 2026 Zevra Therapeutics, Inc. has announced that the European Medicines Agency’s (EMA) Committee for Medicinal...
Minoryx Therapeutics Secures CHMP Backing for NEZGLYAL® in cALD
Barcelona, Spain and Düsseldorf, Germany – July 24, 2026 Minoryx Therapeutics and Neuraxpharm Group have received a positive opinion...
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