Journal of Genetic Syndromes & Gene Therapy
Open Access

4^th World Congress on Rare Diseases and Orphan Drugs

June 11-12, 2018 | Dublin, Ireland

Posters & Accepted Abstracts

• Adherence to antimycetomal medications and factors that may affect it

  Duaa Mohammadat

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• Large-single scale mitochondrial DNA deletions in different tissues of patients with Kearns-Sayre syndrome

  Yuqing shi,

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• Paracrine action of human mesenchymal stem cells for muscle diseases

  Jong Wook Chang

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• Hereditary spastic paraplegias phenotype constitute part of broader rare genetic Mendelian inherited disorders

  Alice Abdel Aleem, Mahmoud F Elsaid, Nader Chalhoub, Khalid Ibrahim, MajidKamli, Firas AlTae, Omer F Kuzu and Arun Sikaruar

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• Patients without diagnosis: A profile

  Isabelle C Windheuser, Martin Muecke, Frank Klawonn and Christiane Stieber

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• Biochemical and electron microscopic changes induced by Giardia in experimentally infected lambs

  Mervat E I Radwan

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• Neglected tropical diseases: Road to control, elimination and eradication by 2020

  Diego-Abelardo Alvarez Hernandez

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• Prevalence and outcome of Treatable lysosomal storage diseases in Children from north- eastern part of Libya

  Professor Nuri M. Shembesh, Dr.abdulsallam A. Shakmak, DR.Fauzia Fazani, Dr.Amal Elwarfale and Dr.Salma Elaubedee

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• Consulting for a combination of molecular defects for variable expression

  Yingjun Xie, Haiming Yuan, and Xiaofang Sun

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• Rapid progression osteolysis in Gorham Stout Syndrome. A case report and literature review.

  Ivan Cherrez Ojeda MD, Leonardo Cano Cevallos MD and Andrea Velez Vera MD

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• From promising molecules to orphan drugs: Early clinical drug development

  Marc Dooms

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• The safety and efficacy of prednisolone in preventing re-accumulation of ascites among endomyocardial fibrosis patients in Uganda: A randomised clinical trial

  Yvonne Brenda Nabunnya, James Kayima, Juergen Freers, Chris T Longenecker and Richard A Josephson

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• Streptococcus suis: Bacteremia presenting with Fever, Rashes, Arthritis and Neurologic Deficits

  Ahmad M. Domado

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• Development of orphan medical products

  Martine Zimmermann

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• Ion transport defects in Microvillus Inclusion Disease (MVID)

  Nadia Ameen

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• Current challenges on Chagas disease

  Jorge-Alberto Ascencio-Aragon

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• Management of rare diseases: An integrated approach to break down barriers and facilitate patient access to healthcare

  Gayathri Balasubramanian

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• 45,X/46,X,i (X) (q10) isochromosome Xq in Mosaic Turner syndrome: A case report

  Mark Ramon Victor B Llanes and May Uyking-Naranjo

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• Sclerosing Angiomatoid Nodular Transformation of the Spleen: An Unusual finding in Paediatric Population

  Godwin Oligbu, Indula Bopitiya, Praveen Saroey, Atra Ayad

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• Drug repurposing screen for rapid identification of therapeutics for Zika virus infection

  Wei Zheng

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• Retrodifferentiation in the treatment of a rare condition: Acquired aplastic anaemia

  Ilham Abuljadayel

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• 45,X / 46,X,i (X) (q10) Isochromosome Xq in mosaic turner syndrome: A case report

  Mark Ramon Victor B. Llanes, MD, May Uyking-Naranjo

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