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Epilepsy & behavior : E&B, ISSN 1525-5050, 01/2020, Volume 107, p. 106866
Journal Article
NATURE REVIEWS DRUG DISCOVERY, ISSN 1474-1776, 07/2019, Volume 18, Issue 7, pp. 479 - 480
Journal Article
ORPHANET JOURNAL OF RARE DISEASES, ISSN 1750-1172, 06/2019, Volume 14, Issue 1, pp. 154 - 7
Journal Article
Journal Article
Journal Article
Neuromuscular Disorders, ISSN 0960-8966, 07/2019, Volume 29, Issue 7, pp. 514 - 516
•Outcome measures in trials for ambulant DMD patients are the 6 MWT, NSAA and 4SC.•Limitations related to patient motivation during the assessment for these... 
European regulators | Clinical trial endpoints | Duchenne muscular dystrophy | Gait Variables | 6-MINUTE WALK TEST | NEUROSCIENCES | CLINICAL NEUROLOGY | Measurement | Medical equipment | Physiological apparatus
Journal Article
Drug Discovery Today, ISSN 1359-6446, 02/2020, Volume 25, Issue 2, pp. 274 - 291
•Non-clinical animal models for rare infectious conditions are reviewed.•The article on infections which are either eradicated or very rare in the EU.•COMP... 
NONHUMAN PRIMATE MODELS | IN-VITRO | INFLUENZA-A VIRUS | H5N1 VIRUS | AVIAN INFLUENZA | MONKEYPOX VIRUS-INFECTION | ANIMAL-MODELS | CYNOMOLGUS MACAQUES | BACILLUS-ANTHRACIS | PHARMACOLOGY & PHARMACY | VISCERAL LEISHMANIASIS
Journal Article
Drug Discovery Today, ISSN 1359-6446, 01/2018, Volume 23, Issue 1, pp. 90 - 100
•The concept of significant benefit stems from the orphan regulation in Europe.•Significant benefit is a clinical advantage and/or a major contribution to... 
PRODUCTS | PHARMACOLOGY & PHARMACY | Orphan Drug Production | Drug Approval | European Union | Europe | Humans
Journal Article
Journal Article
Journal Article
The EMBO Journal, ISSN 0261-4189, 04/1996, Volume 15, Issue 7, pp. 1650 - 1657
The Neurospora crassa blind mutant white collar‐1 (wc‐1) is pleiotropically defective in all blue light‐induced phenomena, establishing a role for the wc‐1... 
Neurospora crassa | Blue light | Zinc finger domain | Photomorphogenesis | PATHWAYS | DNA-BINDING DOMAIN | Neurospora crassal | BIOCHEMISTRY & MOLECULAR BIOLOGY | TRANSCRIPTION | blue light | zinc finger domain | CELL BIOLOGY | photomorphogenesis | MUTANTS | SEQUENCE | CRASSA | PHOTOREGULATION | CAROTENOID BIOSYNTHETIC GENE | EXPRESSION | PHYTOCHROME
Journal Article
Translational Oncology, ISSN 1936-5233, 09/2009, Volume 2, Issue 3, pp. 157 - 163
Liver cancers in children are rare representing only 1.1% of malignancies, with an annual incidence rate of 1.5 cases per million. Hepatoblastoma and... 
HEPATOCELLULAR-CARCINOMA | METHYLATION | PROFILES | MEMBERS | SUPPRESSOR GENE | ONCOLOGY | PROLIFERATION | OVARIAN-CANCER | BIRTH CHARACTERISTICS | IDENTIFICATION | CLUSTER
Journal Article
Nature reviews. Drug discovery, 09/2018
The definition and acceptability of an orphan condition is pivotal for the assessment of European orphan medicinal product designation applications, and... 
Journal Article