Results 101 to 110 of about 270,959 (189)

Correction to: Sharing is caring: a call for a new era of rare disease research and development

Orphanet Journal of Rare Diseases, 2023
Nathan Denton, Andrew E. Mulberg, Monique Molloy, Samantha Charleston, David C. Fajgenbaum, Eric D. Marsh, Paul Howard   +6 more
doaj   +1 more source

Acquiring orphans

Nature Biotechnology, 2014
Rooswinkel, R., Mulder, G.J., Deventer, S. van   +2 more
openaire   +4 more sources

Medicine Development and Access for Rare Diseases: Can We Do Better? [PDF]

J Inherit Metab Dis
Hollak CEM, Rosenberg N, Post C, Stolwijk N, Manders E, Schoenmakers D, Penninx B, Reijnhout N, Verdeyen K, Jonker R, Bosch AM, Langeveld M, van der Wel V, de Visser S, van den Berg S.   +14 more
europepmc   +1 more source

Safety population size and duration of exposure prior to approval of new medicines: A database analysis of medicines centralised approved in the European Union between 2011 and 2023. [PDF]

PLoS One
Bouwman L, Leufkens H, Sepodes B, Torre C.   +3 more
europepmc   +1 more source

Orphan Embryos [PDF]

, 1984
Connery, John R.
core   +1 more source

Orphan medical devices: addressing the regulatory and access gaps in the EU and US. [PDF]

Front Public Health
Akodad S, Haon B, Rochaix L, Stevens H.
europepmc   +1 more source

Candidate orphan genes: Reassessing uniqueness. [PDF]

PLoS One
Maan SK, Butzin XY, Ge SX, Butzin NC.
europepmc   +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

Trends, lag and characteristics of rare disease drug approval in the USA and China, 1983-2022. [PDF]

Orphanet J Rare Dis
Wang S, Liu X, Zou Y, Tang Y, Zuo W, Jiang R, Shang J, Tian X, Liu Q, Xu T, Zhang B, Zhang S.   +11 more
europepmc   +1 more source

Methodological challenges in Dutch HTA of non-oncological orphan drugs: a retrospective analysis and price comparison using different pricing models. [PDF]

Orphanet J Rare Dis
Walraven J, Kaveh M, Uyl-de Groot C.
europepmc   +1 more source