Journal of Genetic Syndromes & Gene Therapy
Open Access

4^th World Congress on Rare Diseases and Orphan Drugs

June 11-12, 2018 | Dublin, Ireland

Scientific Tracks Abstracts

• Rare diagnosis in disorders/differences of sex development

  Yolande van Bever

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• Evaluating the impact of peer support and connection on the quality of life of patients with familial chylomicronemia syndrome (FCS)

  Gilstrap A, Salvatore V, Williams K, Hsieh A, Gwosdow AR, Stevenson M, Davidson D

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• Pulmonary arterial hypertension and pulmonary fibrosis: Treatments, unmet needs, and future directions

  Laxminarayan Bhat

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• Synthetic messenger RNA-based therapeutic strategy for treatment of alpha-1-antitrypsin deficiency

  Tatjana Michel

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• Surgical treatment of scoliosis in rare diseases: Arthogryposis

  Gianluca Colella, Tiziana Greggi, Konstantinos Martikos and Francesco Vommaro

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• Results of the investigation of findings and observations captured in burden of illness survey in FCS patients (in-focus) study: European respondents

  Williams KR, Davidson M, Stevenson M, Hsieh A, Jones R, Issa B, Romeo S

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• Hepatitis c virus testing and treatment among persons receiving buprenorphine in an office-based program for opioid use disorders in Nigeria

  Samuel Ayoola Abati

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• Synthetic messenger RNA (MRNA) as a therapeutic tool

  Tatjana Michel

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• Rare diseases in west Africa

  Mady Ndiaye, Oumar Faye, Mamadou Lamine Djiba and Massamba Sylla

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