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Academic Journal

Optimus-Era Dose Finding for Rare Cancers.

Subjects: Neoplasms*/Neoplasms*/Neoplasms*/drug therapy ; Neoplasms*/Neoplasms*/Neoplasms*/diagnosis ; Rare Diseases*/Rare Diseases*/Rare Diseases*/drug therapy

  • Source: Cancer discovery [Cancer Discov] 2024 Jun 03; Vol. 14 (6), pp. 909-914.Publisher: American Association for Cancer Research Country of Publication: United States NLM ID: 101561693 Publication Model:

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Academic Journal

Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach.

  • Authors : Adang LA; Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA. Electronic address: .; Sevagamoorthy A

Subjects: Rare Diseases*/Rare Diseases*/Rare Diseases*/diagnosis ; Rare Diseases*/Rare Diseases*/Rare Diseases*/therapy ; Rare Diseases*/Rare Diseases*/Rare Diseases*/epidemiology

  • Source: Molecular genetics and metabolism [Mol Genet Metab] 2024 May; Vol. 142 (1), pp. 108453. Date of Electronic Publication: 2024 Mar 18.Publisher: Academic Press Country of Publication: United States NLM ID: 9805456 Publication Model: Print-Electronic Cited

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Academic Journal

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

  • Authors : Mease C; Office of Orphan Products Development, Office of the Commissioner, US Food and Drug Administration, 10903 New Hampshire Ave, Silver Spring, MD, 20993, USA. .; Miller KL

Subjects: Neoplasms*/Neoplasms*/Neoplasms*/drug therapy ; Rare Diseases*/Rare Diseases*/Rare Diseases*/drug therapy; Child

  • Source: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Feb 25; Vol. 19 (1), pp. 86. Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172

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Academic Journal

Navigating Pathways to Diagnosis in Idiopathic Subglottic Stenosis: A Qualitative Study.

  • Authors : Damico Smith C; Department of Surgery, University of Wisconsin-Madison, Madison, Wisconsin, U.S.A.; Nanda N

Subjects: Rare Diseases* ; Laryngostenosis*/Laryngostenosis*/Laryngostenosis*/diagnosis; Humans

  • Source: The Laryngoscope [Laryngoscope] 2024 Feb; Vol. 134 (2), pp. 815-824. Date of Electronic Publication: 2023 Sep 23.Publisher: Wiley-Blackwell Country of Publication: United States NLM ID: 8607378 Publication Model: Print-Electronic Cited

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Academic Journal

Determining Commonalities in the Experiences of Patients with Rare Diseases: A Qualitative Analysis of US Food and Drug Administration Patient Engagement Sessions.

  • Authors : Mease C; Office of Orphan Products Development, Office of the Commissioner, US Food and Drug Administration, 10903 New Hampshire Ave, Silver Spring, MD, 20993, USA. .; Fermaglich LJ

Subjects: Rare Diseases*/Rare Diseases*/Rare Diseases*/psychology ; Quality of Life*/Quality of Life*/Quality of Life*/psychology; Humans

  • Source: The patient [Patient] 2024 Jan; Vol. 17 (1), pp. 25-37. Date of Electronic Publication: 2023 Oct 13.Publisher: Adis, Springer International Country of Publication: New Zealand NLM ID: 101309314 Publication Model: Print-Electronic Cited Medium: Internet

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Academic Journal

Pharmaceutical policy and innovation for rare diseases: A narrative review.

  • Authors : Alonso Ruiz A; Global Health Centre, Graduate Institute of International and Development Studies, Geneva, 1211, Switzerland.; Large K

Subjects: Rare Diseases*/Rare Diseases*/Rare Diseases*/drug therapy ; Orphan Drug Production*/Orphan Drug Production*/Orphan Drug Production*/legislation & jurisprudence; Humans

  • Source: F1000Research [F1000Res] 2023 Nov 13; Vol. 12, pp. 211. Date of Electronic Publication: 2023 Nov 13 (Print Publication: 2023).Publisher: F1000 Research Ltd Country of Publication: England NLM ID: 101594320 Publication Model: eCollection Cited Medium: Internet ISSN:

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Academic Journal

Increasing orphan drug loss in Japan: Trends and R&D strategy for rare diseases.

  • Authors : Enya K; Graduate School of Design and Architecture, Nagoya City University, Nagoya 464-0083, Japan.; Lim Y

Subjects: Orphan Drug Production* ; Rare Diseases*/Rare Diseases*/Rare Diseases*/drug therapy; Humans

  • Source: Drug discovery today [Drug Discov Today] 2023 Oct; Vol. 28 (10), pp. 103755. Date of Electronic Publication: 2023 Aug 28.Publisher: Elsevier Science Ltd. Country of Publication: England NLM ID: 9604391 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Academic Journal

Rare Disease, Advocacy and Justice: Intersecting Disparities in Research and Clinical Care.

Subjects: Rare Diseases* ; Social Justice*; Humans

  • Source: The American journal of bioethics : AJOB [Am J Bioeth] 2023 Jul; Vol. 23 (7), pp. 17-26. Date of Electronic Publication: 2023 May 19.Publisher: Taylor & Francis Country of Publication: United States NLM ID: 100898738 Publication Model: Print-Electronic Cited

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Academic Journal

Implementation of genomic medicine for rare disease in a tertiary healthcare system: Mayo Clinic Program for Rare and Undiagnosed Diseases (PRaUD).

  • Authors : Pinto E Vairo F; Center for Individualized Medicine, Mayo Clinic, 200 First Street SW, Rochester, MN, 55905, USA.; Department of Clinical Genomics, Mayo Clinic, Rochester, MN, USA.

Subjects: Rare Diseases*/Rare Diseases*/Rare Diseases*/diagnosis ; Rare Diseases*/Rare Diseases*/Rare Diseases*/genetics ; Rare Diseases*/Rare Diseases*/Rare Diseases*/therapy

  • Source: Journal of translational medicine [J Transl Med] 2023 Jun 23; Vol. 21 (1), pp. 410. Date of Electronic Publication: 2023 Jun 23.Publisher: BioMed Central Country of Publication: England NLM ID: 101190741 Publication Model: Electronic Cited Medium: Internet ISSN: 1479-5876

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Academic Journal

Exploring the Potential Challenges for Developing Generic Orphan Drugs for Rare Diseases: A Survey of US and European Markets.

Subjects: Orphan Drug Production* ; Rare Diseases*/Rare Diseases*/Rare Diseases*/drug therapy; United States

  • Source: Value in health regional issues [Value Health Reg Issues] 2023 May; Vol. 35, pp. 87-94. Date of Electronic Publication: 2023 Mar 13.Publisher: Elsevier Country of Publication: United States NLM ID: 101592642 Publication Model: Print-Electronic Cited Medium:

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  • 1-10 of  1,284 results for ""Rare Diseases""