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Duchenne Muscular Dystrophy
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Information and abstracts for author publications
Nature reviews. Drug discovery
Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years.
Cell stem cell
The Orphan Drug Act and the development of stem cell-based products for rare diseases.
Clinical pharmacology and therapeutics
The impact of the Orphan Drug Act on the development and advancement of neurological products for rare diseases: a descriptive review.
Briefings in bioinformatics
Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseases.
Therapies for inborn errors of metabolism: what has the orphan drug act delivered?
Muscle & nerve
Duchenne muscular dystrophy: Drug development and regulatory considerations.
Preparation of Chloroplast Sub-compartments from Arabidopsis for the Analysis of Protein Localization by Immunoblotting or Proteomics
Preparation of Chloroplast Sub…
Capture and Identification of RNA-binding Proteins by Using Click Chemistry-assisted RNA-interactome Capture (CARIC) Strategy
Capture and Identification of …
Model for the Evaluation of Endoscopic Submucosal Injection Material Performance
Time-lapse 3D Imaging of Phagocytosis by Mouse Macrophages
Time-lapse 3D Imaging of Phago…
Approaches to Determine Intestinal Epithelial Cell Permeability
Real-time Imaging and Quantification of Fungal Biofilm Development Using a Two-Phase Recirculating Flow System
Real-time Imaging and Quantifi…
Eye-tracking to Distinguish Comprehension-based and Oculomotor-based Regressive Eye Movements During Reading
Eye-tracking to Distinguish Co…
Adult Zebrafish Injury Models to Study the Effects of Prednisolone in Regenerating Bone Tissue
Adult Zebrafish Injury Models …
Two-photon Imaging of Cortical Neurons in Neonatal Mice
Contractions of Human-iPSC-derived Cardiomyocyte Syncytia Measured with a Ca-sensitive Fluorescent Dye in Temperature-controlled 384-well Plates
Contractions of Human-iPSC-der…
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