Results 101 to 110 of about 109,656 (184)

Correction to: Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development

Orphanet Journal of Rare Diseases
Catherine Mease, Kathleen L. Miller, Lewis J. Fermaglich, Jeanine Best, Gumei Liu, Erika Torjusen   +5 more
doaj   +1 more source

Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA. [PDF]

Ther Adv Respir Dis
Höger P, Ries M, Olivares Rivera A, Ersöz H, Buschulte K, Fähndrich S, Kontogianni K, Herth F, Trudzinski FC.   +8 more
europepmc   +1 more source

Advances in orphan drug development: Time to change the status and stereotype. [PDF]

Perspect Clin Res, 2022
Thakur S.
europepmc   +1 more source

Determinants of orphan drug health technology assessment in South Korea: an empirical analysis. [PDF]

Front Pharmacol
Lee SH, Lee SK, Lee JH, Bang JS.
europepmc   +1 more source

Concurrent nanotherapeutics and regulatory updates for the management of amyotrophic lateral sclerosis: a focused review for orphan drug (Tofersen). [PDF]

Orphanet J Rare Dis
Kumar A, Shukla S, Rai A, Pathak P, Narayan KP.   +4 more
europepmc   +1 more source

How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis. [PDF]

Orphanet J Rare Dis, 2023
Frost J, Hall A, Taylor E, Lines S, Mandizha J, Pope C.   +5 more
europepmc   +1 more source

Trends in orphan drug use and spending among children and adolescents during 2010-2020 in Korea. [PDF]

Front Pharmacol, 2022
Oh JY, Lee JY, Kim DS.
europepmc   +1 more source

Is It Possible to Have Cheaper Drugs and Preserve the Incentive to Innovate: Reforming the Drug Approval Process According to Market Principles [PDF]

This paper argues that drugs are expensive not because of a lack of competition among research-based pharmaceutical companies, but because of a lack of competition in the drug approval process.
Corinne Sauer, Robert Sauer
core  

Orphan drug uttroside B impedes MASH progression and HCC development in experimental models. [PDF]

JHEP Rep
Rayginia TP, Keerthana CK, Aiswarya SU, Shifana SC, Jannet S, Varma SS, Maria Joy P, Swetha M, James S, Aparna JS, Vijayan Y, Retnakumary AP, Nath LR, Kalimuthu K, Jaikumar VS, Sundaram S, Anto NP, Isakov N, Harikumar KB, Lankalapalli RS, Anto RJ.   +20 more
europepmc   +1 more source

The impact of the rare disease and Orphan Drug Act in Taiwan. [PDF]

J Food Drug Anal, 2021
Hsiang NC, Huang WF, Gau CS, Tsai TW, Chang LC.   +4 more
europepmc   +1 more source