Clinical Pharmacology in Drug Development for Rare Diseases in Neurology: Contributions and Opportunities

Innovative design and analysis for rare disease drug development

(2020) Shein-Chung Chow et al.   Journal of Biopharmaceutical Statistics

Huntington's Disease Progression: A Population Modeling Approach to Characterization Using Clinical Rating Scales

(2020) Wan Sun et al.   JOURNAL OF CLINICAL PHARMACOLOGY

Artificial Intelligence and Machine Learning Applied at the Point of Care

(2020) Zuzanna Angehrn et al.   Frontiers in Pharmacology

Pathogenesis and therapeutic targets in spinal muscular atrophy (SMA)

(2020) S. Lefebvre et al.   ARCHIVES DE PEDIATRIE

Artificial Intelligence (AI) in Rare Diseases: Is the Future Brighter?

(2019) Sandra Brasil et al.   Genes

A pharmaco-metabolomics approach in a clinical trial of ALS: Identification of predictive markers of progression

(2018) Hélène Blasco et al.   PLoS One

Flexibility in the FDA approach to orphan drug development

(2017) Nina L. Hunter et al.   NATURE REVIEWS DRUG DISCOVERY

Elvitegravir/Cobicistat/Tenofovir/Emtricitabine Penetration in the Cerebrospinal Fluid of Three HIV-Positive Patients

(2016) Andrea Calcagno et al.   AIDS RESEARCH AND HUMAN RETROVIRUSES

Challenges and Opportunities in Rare Disease Drug Development

(2016) BP Smith   CLINICAL PHARMACOLOGY & THERAPEUTICS

Accelerated Approval Paths: What they do Mean and What they Should Not Mean?

(2016) G. Calvo   CLINICAL THERAPEUTICS

Important Role of Translational Science in Rare Disease Innovation, Discovery, and Drug Development

(2014) Anne R. Pariser et al.   JOURNAL OF GENERAL INTERNAL MEDICINE

Using Simcyp to project human oral pharmacokinetic variability in early drug research to mitigate mechanism-based adverse events

(2011) Christopher L. Shaffer et al.   BIOPHARMACEUTICS & DRUG DISPOSITION

Oligonucleotide therapeutic approaches for Huntington disease

(2011) Dinah W.Y. Sah et al.   JOURNAL OF CLINICAL INVESTIGATION

The Impact of the Orphan Drug Act on the Development and Advancement of Neurological Products for Rare Diseases: A Descriptive Review

(2010) K A Burke et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Biomarkers in Huntington's and Parkinson's Disease

(2009) Gráinne C. O’Keeffe et al.   Annals of the New York Academy of Sciences

The FDA Critical Path Initiative and Its Influence on New Drug Development

(2008) Janet Woodcock et al.   Annual Review of Medicine

Clinical research for rare disease: Opportunities, challenges, and solutions

(2008) Robert C. Griggs et al.   MOLECULAR GENETICS AND METABOLISM