Results 181 to 190 of about 8,062,145 (231)

Small molecule inhibitor of orphan GPCR dimerization improves host defense and blood pressure control in mice. [PDF]

J Clin Invest
Kwon J, Persechino M, Shao J, Shamsara J, Spitznagel B, Salwig I, Sanda M, Offermanns S, Kolb P, Wettschureck N.   +9 more
europepmc   +1 more source

Optimizing regional rare disease management systems within China's policy framework: practical insights from the shantou experience. [PDF]

Orphanet J Rare Dis
Wu B, Wang Y, Liu C, Wang Q, Gong M, Chen Y.   +5 more
europepmc   +1 more source

Issues Surrounding Orphan Disease and Orphan Drug Policies in Europe

An orphan disease is a disease with a very low prevalence. Although there are 5000-7000 orphan diseases, only 50 orphan drugs (i.e. drugs developed to treat orphan diseases) were marketed in the EU by the end of 2008. In 2000, the EU implemented policies
Lut Mergaert, Christel Fostier, Steven Simoens, Alain Denis, Irina Cleemput   +4 more
core  

Size Does Matter! Prioritizing Rare Diseases for Luck Egalitarian Reasons. [PDF]

Bioethics
Andersen DB, Albertsen AB, Wadmann S, Bech M.   +3 more
europepmc   +1 more source

Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study. [PDF]

Orphanet J Rare Dis
Antoni G, Kumps C, Berger J, Barman-Aksözen J, Tran C.   +4 more
europepmc   +1 more source

Healthy Participant Versus Patient Selection for Clinical Pharmacology Studies in Small‐Molecule Oncology Drug Development

Clinical and Translational Science, Volume 19, Issue 11, November 2026.
Paridhi Gupta, Zheyi Hu, Li Zhu, Lora Hamuro   +3 more
wiley   +1 more source

Current Advances in Liver-Targeted Drug Delivery: Synthetic, Biological, and Biomimetic Platforms. [PDF]

Int J Mol Sci
Sergazy S, Seidakhmetova RB, Zakirov Y, Zhilkaidarov A, Amirbek D, Shulgau Z, Berikkhanova K, Gulyaev A.   +7 more
europepmc   +1 more source

Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies. [PDF]

J Mark Access Health Policy
Blandy O, Lembo P, Folorunso R, Myren KJ, Chevrou-Severac H, Thomas SK.   +5 more
europepmc   +1 more source

Ras-MAPK somatic variants in mesial temporal lobe epilepsy. [PDF]

Curr Opin Genet Dev
Cono S, Kahle KT, Walsh CA, Khoshkhoo S.
europepmc   +1 more source

Role of Bile Acid Receptors in Non-Infectious Diarrhea: A Narrative Review. [PDF]

Med Sci Monit
Pan L, Ran P, Jiang F, Wang Y, Wang W, Zhao R, Mu H, Fu G.   +7 more
europepmc   +1 more source