Literature DB >> 31942878

10th World Orphan Drug Congress (WODC) (November 12-14, 2019 - Barcelona, Spain).

K Künnemann1.   

Abstract

The 10th World Orphan Drug Congress (WODC), now recognized as the largest and most established European orphan drug event, took place once again November 12-14, 2019, in Barcelona, Spain. Like in previous years, the more than 600 attendees were composed of government authorities, payers, industry and patient advocacy groups as well as biotech start-ups and investors. The 2019 congress aimed to address the strategic and commercial aspects of bringing new treatments to rare disease patients. The more than 200 speakers discussed many different rare disease aspects for clinical and product development, market access and pricing, manufacture, science and strategy, and precision medicine. A co-conference on Cell and Gene Therapy was also organized. This rare disease conference addressed many different challenges in the field with numerous discussions on how to improve cross-border communications, how to better identify patients and shorten their diagnosis time using new tools such as artificial intelligence and machine learning, as well as how to improve usage of patient data and patient empowerment. Copyright 2019 Clarivate Analytics.

Entities:  

Keywords:  AVR-RD-01; AVR-RD-02; AVR-RD-03; AVR-RD-04; Artificial intelligence; Gaboxadol; HMI-102; Machine learning; Patient advocacy groups

Year:  2019        PMID: 31942878     DOI: 10.1358/dot.2019.55.12.3107705

Source DB:  PubMed          Journal:  Drugs Today (Barc)        ISSN: 1699-3993            Impact factor:   2.245


  1 in total

1.  Ceria-Zirconia nanoparticles reduce intracellular globotriaosylceramide accumulation and attenuate kidney injury by enhancing the autophagy flux in cellular and animal models of Fabry disease.

Authors:  Jong Hun An; Sang-Eun Hong; Seong-Lan Yu; Jaeku Kang; Chang Gyo Park; Hoi Young Lee; Sung-Ki Lee; Dong Chul Lee; Hwan-Woo Park; Won-Min Hwang; Sung-Ro Yun; Yohan Park; Moon Hyang Park; Kuk Ro Yoon; Se-Hee Yoon
Journal:  J Nanobiotechnology       Date:  2022-03-09       Impact factor: 10.435

  1 in total

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