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38,449 results for “Orphan Drug”

Trials

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Orphan Drugs for Inherited Metabolic Diseases

39 participants · First posted Apr 19, 2023

Reversing External-beam Radiotherapy-associated Fibrosis Syndrome: an Interventional Bayesian Adaptive Randomized-controlled Orphan Drug Platform Trial for Orodental Sequelae (Reverse-fibrose)

250 participants · First posted Apr 6, 2025

Active, not recruiting

Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

7 participants · First posted Aug 28, 2019

Drugs

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Antibody oligonucleotide conjugate (AOC)

Investigational

AOC-1020(del-brax, delpacibart braxlosiran)

Manufacturer: Novartis

GPCR modulators (orphan neurological disorder)

Elsilimomab(AOP Orphan Pharmaceuticals AG)

Organizations

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argenx SE

Belgium

Cadrenal Therapeutics

United States

Wuhan Healthgen Biotechnology Corp.

News

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Japan Emerges as Second-Choice Market for Orphan Drug Launches, New Analysis Reveals

Orphan Drug Market Growth Slowing but Remains Strategic Priority for Pharma

US Orphan Drug Market Poised to Exceed $190 Billion by 2030 as FDA Designations Accelerate Rare Disease Therapy Development

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