Rare Diseases
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Published By Landes Bioscience

2167-5511, 2167-549x
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scholarly journals Simvastatin offers new prospects for the treatment of Duchenne muscular dystrophy

Rare Diseases ◽  
2016 ◽  
Vol 4 (1) ◽  
pp. e1156286 ◽  
Author(s):  
Nicholas P. Whitehead ◽  
Min Jeong Kim ◽  
Kenneth L. Bible ◽  
Marvin E. Adams ◽  
Stanley C. Froehner
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy

scholarly journals Beyond multiple mechanisms and a unique drug: Defective autophagy as pivotal player in cerebral cavernous malformation pathogenesis and implications for targeted therapies

Rare Diseases ◽  
2016 ◽  
Vol 4 (1) ◽  
pp. e1142640 ◽  
Author(s):  
Saverio Marchi ◽  
Eliana Trapani ◽  
Mariangela Corricelli ◽  
Luca Goitre ◽  
Paolo Pinton ◽  
...  
Keyword(s):  
Targeted Therapies ◽  
Cavernous Malformation ◽  
Cerebral Cavernous Malformation

scholarly journals Neurodevelopmental models of transcription factor 4 deficiency converge on a common ion channel as a potential therapeutic target for Pitt Hopkins syndrome

Rare Diseases ◽  
2016 ◽  
Vol 4 (1) ◽  
pp. e1220468 ◽  
Author(s):  
Matthew D. Rannals ◽  
Stephanie Cerceo Page ◽  
Morganne N. Campbell ◽  
Ryan A. Gallo ◽  
Brent Mayfield ◽  
...  
Keyword(s):  
Transcription Factor ◽  
Ion Channel ◽  
Therapeutic Target ◽  
Potential Therapeutic Target ◽  
Common Ion ◽  
Transcription Factor 4

scholarly journals Canine models of human rare disorders

Rare Diseases ◽  
2016 ◽  
Vol 4 (1) ◽  
pp. e1241362 ◽  
Author(s):  
Marjo K. Hytönen ◽  
Hannes Lohi
Keyword(s):  
Rare Disorders ◽  
Canine Models

scholarly journals Dystrophin: The dead calm of a dogma

Rare Diseases ◽  
2016 ◽  
Vol 4 (1) ◽  
pp. e1153777 ◽  
Author(s):  
Dariusz C. Górecki
Keyword(s):  
The Dead

scholarly journals Pathological relationships involving iron and myelin may constitute a shared mechanism linking various rare and common brain diseases

Rare Diseases ◽  
2016 ◽  
Vol 4 (1) ◽  
pp. e1198458 ◽  
Author(s):  
Moones Heidari ◽  
Sam H. Gerami ◽  
Brianna Bassett ◽  
Ross M. Graham ◽  
Anita C.G. Chua ◽  
...  
Keyword(s):  
Brain Diseases

scholarly journals The calpain-suppressing effects of olesoxime in Huntington's disease

Rare Diseases ◽  
2016 ◽  
Vol 4 (1) ◽  
pp. e1153778 ◽  
Author(s):  
Jonasz J. Weber ◽  
Midea M. Ortiz Rios ◽  
Olaf Riess ◽  
Laura E. Clemens ◽  
Huu P. Nguyen
Keyword(s):  
Huntington's Disease ◽  
Huntington’S Disease
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