نتائج البحث

Development of orphan drugs for rare diseases.

• Authors : Han-Wook Yoo¹

• Source: Clinical & Experimental Pediatrics. Jul2024, Vol. 67 Issue 7, p315-327. 13p.

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Efficacy and safety of compassionate use for rare diseases: a scoping review from 1991 to 2022.

• Authors : Wu, Jiayu¹ (AUTHOR); Yang, Yang¹ (AUTHOR); Yu, Jiaxin¹ (AUTHOR)

• Source: Orphanet Journal of Rare Diseases. 11/28/2023, Vol. 18 Issue 1, p1-14. 14p.

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Enfermedades raras en un servicio de genética médica de población con seguridad social.

• Authors : Jiménez-Pérez, Berenice¹; Juárez-Melchor, Daniela¹ ; Alejandra Guzmán-Santiago, Tania¹

• Source: Revista Medica del IMSS. may/jun2024, Vol. 62 Issue 3, p1-8. 8p.

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Regulatory Overview on Rare Diseases and Orphan Drugs in India.

• Authors : M. S., JYOTHI¹ ; V., BALAMURALIDHARA² ; S., NAGARAJU¹

• Source: International Journal of Pharmaceutical Research (09752366). 2020 Supplement1, p1957-1965. 9p.

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(In)-Equality in the Allocation of R&D Resources for Rare Diseases.

• Authors : Raïs Ali, Setti¹ ; Tubeuf, Sandy^2,3

• Source: Social Justice Research. Sep2019, Vol. 32 Issue 3, p277-317. 41p. 15 Charts, 4 Graphs.

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The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success.

• Authors : SALTONSTALL, PETER; ROSS, HEIDI; KIM, PAUL T.

Subjects: ORPHAN drug laws; CRITICISM; ORPHAN drugsUNITED States

• Source: Milbank Quarterly. Mar2024, Vol. 102 Issue 1, p83-96. 14p.

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Proteomic signatures improve risk prediction for common and rare diseases.

• Authors : Carrasco-Zanini, Julia; Pietzner, Maik; Davitte, Jonathan

Subjects: 32 Biomedical and Clinical Sciences; 3201 Cardiovascular Medicine and Haematology; Rare Diseases

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Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study

• Authors : Francesca Gorini; Michele Santoro; Anna Pierini

Subjects: orphan drug; rare disease; registry

• Source: Frontiers in Pharmacology, Vol 13 (2022)

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Amyotrophic lateral sclerosis caused by hexanucleotide repeat expansions in C9orf72: from genetics to therapeutics

• Authors : Mizielinska, Sarah; Hautbergue, Guillaume M; Gendron, Tania F

Subjects: Biomedical and Clinical Sciences; Neurosciences; Brain Disorders

• Source: The Lancet Neurology. 24(3)

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Financial conflicts among physician speakers at the April 12, 2024 Oncology Drug Advisory Meeting: Who decided that MRD can be a novel regulatory endpoint in myeloma?

• Authors : Carr, Noah J; Haslam, Alyson; Prasad, Vinay

Subjects: Biomedical and Clinical Sciences; Cardiovascular Medicine and Haematology; Rare Diseases

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