Results 111 to 120 of about 42,003 (185)

Rare Diseases and Orphan Drugs. Limited Lists and its Formulation

, 2022
I. Komarov
openalex   +1 more source

Global insight into rare disease and orphan drug definitions: a systematic literature review. [PDF]

BMJ Open
Abozaid GM, Kerr K, Alomary H, Al-Omar HA, McKnight A.   +4 more
europepmc   +1 more source

The Road to Russian Pharmacoeconomics: Understanding the Drivers for Access to High-Value / Orphan Disease Drugs [PDF]

, 2016
E. Subbotina, CA Chowdhury
openalex   +1 more source

From care to cure: a patient engagement framework for rare disease and orphan drug research. [PDF]

Ther Adv Rare Dis
Awada N, Varughese A.
europepmc   +1 more source

Five-Year Sales for Newly Marketed Prescription Drugs With and Without Initial Orphan Drug Act Designation.

JAMA, 2023
Tu SS, Nagar S, Kesselheim AS, Lu Z, Rome BN.   +4 more
europepmc   +1 more source

Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial [PDF]

, 2018
Verena Wally, Alain Hovnanian, Juliette Ly, Hana Bučková, Victoria Brunner, Thomas Lettner, Michael Ablinger, Thomas K. Felder, Peter Hofbauer, Martin Wolkersdorfer, Florian B. Lagler, Wolfgang Hitzl, Martin Laimer, Sophie Kitzmüller, Anja Diem, Johann Bauer   +15 more
openalex   +1 more source

Towards a sustainable rare disease and orphan drug ecosystem in Saudi Arabia: policy insights from a multi-stakeholder workshop. [PDF]

Front Pharmacol
Abozaid GM, Al-Omar HA, Alrabiah A, AlMuaither A, McKnight AJ.   +4 more
europepmc   +1 more source

Combined assessment of early and late-phase outcomes in orphan drug development. [PDF]

Stat Med, 2021
Pateras K, Nikolakopoulos S, Roes KCB.
europepmc   +1 more source

Using In Silico Tools in Repurposing Drugs for Neglected and Orphan Diseases

, 2016
Sean Ekins
openalex   +1 more source

Successes and pitfalls in orphan drug development for sickle cell disease. [PDF]

Blood Adv
Costa E, Isgrò A, de Montalembert M, Leufkens HGM, Ware RE, De Franceschi L.   +5 more
europepmc   +1 more source