3rd Annual Congress on Rare Diseases and Orphan Drugs

October 30-November 01, 2017 San Antonio, USA

ScientificTracks Abstracts

Androl Gynecol: Curr Res
The use of CRISPR/Cas9 to treat hereditary diseases: Duchenne muscular dystrophy, Friedreich’s ataxia and Alzheimer disease

Jacques P Tremblay, Khadija Cherif, Dominique L Ouellet, Benjamen Duchene, Jean-Paul Iyombe, Catherine Gerard, Joel Rousseau and Antoine Guyon

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Androl Gynecol: Curr Res
Integrating informational resources for drug repurposing in rare diseases

Maria Shkrob, Matthew Clark, Anton Yuryev and Rick Thompson

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Androl Gynecol: Curr Res
L-cystine diamides as a novel therapy for cystinuria

Amrik Sahota, Min Yang, David Goldfarb, Michael Ward, Jay A Tischfield and Longqin Hu

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Androl Gynecol: Curr Res
Using quantitative pharmacology to overcome challenges in hematopoietic cell transplantation: Application towards primary immune deficiencies and inborn errors of metabolism

Janel Long-Boyle

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Androl Gynecol: Curr Res
Nephrotic syndrome: Predicting and defining therapeutic outcomes by systems biology omics approaches

Shipra Agrawal

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Androl Gynecol: Curr Res
Porphyrias-An update on the management and unmet clinical needs

Ashwani K Singal

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Androl Gynecol: Curr Res
The Middle East: A mine for orphan drugs development

Tony Zbeidy

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Androl Gynecol: Curr Res
Pharmacological chaperone therapy for neuronopathic Gaucher disease

Aya Narita

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Androl Gynecol: Curr Res
Modeling Li-Fraumeni syndrome by induced pluripotent stem cells

Dung-Fang Lee

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Androl Gynecol: Curr Res
Genomic approaches in modern biotechnology: From discovery, translation to implementation

David Dongliang Ge

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Androl Gynecol: Curr Res
Consumer directed precision health - convergence of multi- omics, environment, life style and behaviors

Praduman Jain

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Androl Gynecol: Curr Res
A case of Hoffman′s syndrome masquerading as pituitary tumor

Arnab Ghosh and Manish Bhartiya

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Androl Gynecol: Curr Res
Cutaneous infarcts and antineutrophil cytoplasm antibodies positive vasculitis in angioimmunoblastic T-cell lymphoma: A rare presentation

Abhish Mohan, Sandeep Kumar and Pankaj Puri

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Androl Gynecol: Curr Res
POEMS syndrome in a 65 years old lady presenting with peripheral neuropathy and recent onset diabetes

Arnab Ghosh and Sanjeevan Sharma

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