The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation (RPDD) to Arbor Biotechnologies' gene editing therapeutic, ABO-101, to treat ...

Ariceum Therapeutics, a private biotech company based in Berlin, has announced that the US Food and Drug Administration (FDA) ...

Ariceum Therapeutics is a private, clinical stage radiopharmaceutical company focused on the diagnosis and precision treatment of certain neuroendocrine and other aggressive, hard-to-treat cancers.

There have been continued calls to reform orphan drug legislation in the US and the EU, particularly the 40-year-old Orphan Drug Act and the European Union's 1999 Orphan Drug Regulation.

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). Per ...

How can orphan drug manufacturers demonstrate value and improve access to drugs? In this first of two articles on the subject, Ewan Bennett outlines ways to achieve reimbursement in countries ...

News, October 24, 2011 Orphan Drug Status for Corneal Cross-Linking in Keratoconus Corneal cross-linking is the first medical therapy available in the US to slow disease progress. News ...

The "Strategic Intelligence: Orphan Designated Drugs" report has been added to ResearchAndMarkets.com's offering.This report consolidates the latest thinking and forecasts around how the healthcare, ...

DUBLIN--(BUSINESS WIRE)--The "Orphan Drugs - Global Strategic Business Report" report has been added to ResearchAndMarkets.com's offering. The global market for Orphan Drugs was estimated at US$ ...

By Daniella Parra March Biosciences was granted an orphan drug designation from the FDA to MB-105, its CD5-targeted CAR-T ...

Orphan Drugs Market Orphan Drugs Market Dublin, Jan. 22, 2025 (GLOBE NEWSWIRE) -- The "Orphan Drugs - Global Strategic Business Report" report has been added to ResearchAndMarkets.com's offering.The ...

A new drug reimbursement principle is seen as a significant departure from value-based pricing. Despite concerns, the new pricing system could increase the availability of some orphan drugs by 35 per ...