The European Medicines Agency has recommended marketing approval for Alyftrek for cystic fibrosis and Attrogy for a form of ...

An FDA-designated orphan drug that can target a key vulnerability in lung cancer shows promise in improving the efficacy of ...

To ensure timely access to life-saving orphan drugs for patients, the government has exempted mandatory sampling and testing ...

The FDA has granted Orphan Drug designation to NS-229 for the treatment of eosinophilic granulomatosis with polyangiitis.

The FDA has granted Orphan Drug designation to BA-102 for the treatment of Phelan-McDermid syndrome, a rare neurodevelopmental disorder.

Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class1 targeted and ...

There are currently no FDA-approved treatments for FAP, a condition characterized by the proliferation of polyps in the colon and/or rectum.

Innorna, a clinical-stage biotechnology company revolutionizing mRNA therapeutics with its innovative lipid nanoparticle (LNP ...

NS-229 is currently being evaluated in a phase 2 trial in patients with eosinophilic granulomatosis with polyangiitis. The Food and Drug Administration (FDA) has granted Orphan Drug designation to ...

HAS GRANTED ORPHAN DRUG DESIGNATION IN THE EUROPEAN UNION FOR AB8939 IN THE TREATMENT OF ACUTE MYELOID LEUKEMIA Paris, April 23, 2025, 6.45pm CET AB Science SA (Euronext - FR0010557264 - AB) announces ...

Rigorous humanized mouse model enhances credibility and translatability of results- Key preclinical data to support FDA Orphan Drug Application- In vitro success signals potential for exosome-based th ...