EDITORIAL
Published on 28 Sep 2022
Editorial: Prevention, diagnosis and treatment of rare disorders
doi 10.3389/fphar.2022.1026064
- 1,454 views
- 1 citation
- Frontiers in Pharmacology
- Drugs Outcomes Research and Policies
- Research Topics
- Prevention, Diagnosis and Treatment of Rare Disorders
Prevention, Diagnosis and Treatment of Rare Disorders
21k
Total downloads
120k
Total views and downloads
Submit your idea
You will be redirected to our submission process.
Articles
ORIGINAL RESEARCH
Published on 02 May 2022
Characteristics of Early Phase Clinical Trials for Rare Cancers: Insights From Interviews With Stakeholders
doi 10.3389/fphar.2022.775217
- 3,699 views
- 3 citations
ORIGINAL RESEARCH
Published on 25 Mar 2022
Defining Molecular Treatment Targets for Bladder Pain Syndrome/Interstitial Cystitis: Uncovering Adhesion Molecules
doi 10.3389/fphar.2022.780855
- 2,369 views
- 4 citations
ORIGINAL RESEARCH
Published on 25 Mar 2022
Patient-Patient Similarity-Based Screening of a Clinical Data Warehouse to Support Ciliopathy Diagnosis
doi 10.3389/fphar.2022.786710
- 2,341 views
- 6 citations
OPINION
Published on 24 Feb 2022
Nanomedicines and Nanosimilars—Why a Robust Centralised Regulatory Framework Is Essential to Enhance Patient Safety
doi 10.3389/fphar.2021.787239
- 2,152 views
- 4 citations
ORIGINAL RESEARCH
Published on 26 Jan 2022
Availability and Accessibility of Orphan Medicinal Products to Patients in Slovakia in the Years 2010–2019
doi 10.3389/fphar.2022.768325
- 3,721 views
- 1 citation
REVIEW
Published on 21 Jan 2022
Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries
doi 10.3389/fphar.2021.750742
- 12,913 views
- 14 citations
PERSPECTIVE
Published on 10 Jan 2022
Treating Rare Diseases in Africa: The Drugs Exist but the Need Is Unmet
doi 10.3389/fphar.2021.770640
- 6,885 views
- 18 citations
REVIEW
Published on 05 Jan 2022
Challenges in Treating Genodermatoses: New Therapies at the Horizon
doi 10.3389/fphar.2021.746664
- 6,923 views
- 11 citations
REVIEW
Published on 21 Dec 2021
Current Drug Repurposing Strategies for Rare Neurodegenerative Disorders
doi 10.3389/fphar.2021.768023
- 15,017 views
- 27 citations
POLICY AND PRACTICE REVIEWS
Published on 16 Dec 2021
Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group)
doi 10.3389/fphar.2021.744532
- 10,485 views
- 27 citations
PERSPECTIVE
Published on 14 Dec 2021
Crowd Funding for Orphan Drugs: The Case of Baby Pia
doi 10.3389/fphar.2021.787342
- 3,616 views
- 3 citations
Published in
Frontiers in Pharmacology
- Drugs Outcomes Research and Policies
Impact
- 120kTopic views
- 92kArticle views
- 21kArticle downloads