Journal of Genetic Syndromes & Gene Therapy
Open Access

2^nd World Congress on Rare Diseases and Orphan Drugs

June 29-30, 2017 London, UK

Scientific Tracks Abstracts

• Development of orphan medical products

  Martine Zimmermann

  PDF HTML

• Ion transport defects in Microvillus Inclusion Disease (MVID)

  Nadia Ameen

  PDF HTML

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

  Wei Zheng

  PDF HTML

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

  Diego-Abelardo Alvarez Hernandez

  PDF HTML

• Treatment of early onset scoliosis associated with rare disease

  Stefano Giacomini, Tiziana Greggi, Elena Maredi, Mario Di Silvestre, Francesco Vommaro, Aristide Morigi, Maria Renata Bacchin, Valerio Pipola and Francesca Pastorelli

  PDF HTML

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

  Ilham Abuljadayel

  PDF HTML

• Current challenges on chagas disease

  Jorge-Alberto Ascencio-Aragon

  PDF HTML

• Pathway-Centric analysis of rare autoinflammatory diseases (AID) and drug repurposing

  Patrick J Tighe

  PDF HTML

• Consulting for a combination of molecular defects for variable expression

  Yingjun Xie, Haiming Yuan and Xiaofang Sun

  PDF HTML

• Paracrine action of human mesenchymal stem cells for muscle diseases

  Jong Wook Chang

  PDF HTML

• Splice modulation therapy for inherited retinal diseases

  Rob W J Collin

  PDF HTML

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

  Gayathri Balasubramanian

  PDF HTML

• The diagnostic challenge of rare diseases: Lack of knowledge or leaking method?

  Shmuel Prints

  PDF HTML

• 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

  PDF HTML

• Allogeneic retrodifferentiated stem cell therapy for the treatment of dystrophic epidermolysis bullosa

  Ilham Abuljadayel

  PDF HTML