scholarly journals Efficacy, tolerability, and retention of fenfluramine for the treatment of seizures in patients with Dravet syndrome: Compassionate use program in Germany

Epilepsia ◽  
2021 ◽  
Author(s):  
Adam Strzelczyk ◽  
Milka Pringsheim ◽  
Thomas Mayer ◽  
Tilman Polster ◽  
Kerstin A. Klotz ◽  
...  
Keyword(s):  
Dravet Syndrome ◽  
Compassionate Use

scholarly journals A zebrafish-centric approach to antiepileptic drug development

2021 ◽  
Vol 14 (7) ◽  
Author(s):  
Scott C. Baraban
Keyword(s):  
Drug Development ◽  
High Throughput ◽  
Drug Screening ◽  
Danio Rerio ◽  
Genetic Manipulation ◽  
Dravet Syndrome ◽  
Developmental Studies ◽  
Compassionate Use ◽  
Larval Zebrafish ◽  
The Common

ABSTRACT Danio rerio (zebrafish) are a powerful experimental model for genetic and developmental studies. Adaptation of zebrafish to study seizures was initially established using the common convulsant agent pentylenetetrazole (PTZ). Larval PTZ-exposed zebrafish exhibit clear behavioral convulsions and abnormal electrographic activity, reminiscent of interictal and ictal epileptiform discharge. By using this model, our laboratory developed simple locomotion-based and electrophysiological assays to monitor and quantify seizures in larval zebrafish. Zebrafish also offer multiple advantages for rapid genetic manipulation and high-throughput phenotype-based drug screening. Combining these seizure assays with genetically modified zebrafish that represent Dravet syndrome, a rare genetic epilepsy, ultimately contributed to a phenotype-based screen of over 3500 drugs. Several drugs identified in these zebrafish screens are currently in clinical or compassionate-use trials. The emergence of this ‘aquarium-to-bedside’ approach suggests that broader efforts to adapt and improve upon this zebrafish-centric strategy can drive a variety of exciting new discoveries.

Defibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study

2000 ◽  
Vol 111 (4) ◽  
pp. 1122-1129 ◽  
Author(s):  
R. Chopra ◽  
J. D. Eaton ◽  
A. Grassi ◽  
M. Potter ◽  
B. Shaw ◽  
...  
Keyword(s):  
Occlusive Disease ◽  
Compassionate Use

Compassionate Use of Simvastatin for a Patient with Juvenile Metachromatic Leukodystrophy

2017 ◽  
Vol 48 (S 01) ◽  
pp. S1-S45
Author(s):  
A. Bley ◽  
U. Löbel ◽  
J. Denecke
Keyword(s):  
Metachromatic Leukodystrophy ◽  
Compassionate Use
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