Ataxia Telangiectasia; rare genetic disease; IEDAT; Erydel; pivotal phase III study; H2020; clinical study
a pivotal phase III study will be conducted, and regulatory filing for market authorization of the therapy in EU and USA will be made by 2019.
Creating an awareness of Sanfilippo Syndrome and to raise funds to find a cure for this rare genetic disease.
Toute l'actualité Xbox, les tests et bons plans sur Xbox One et Xbox Series X. Infos sur les jeux Xbox, le Xbox Game Pass et le Xbox Live 7j/7.
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The ERS-education website provides centralised access to all educational material produced by the European Respiratory Society. It is the world's largest CME collection for lung diseases and treatment offering high quality e-learning and teaching resources for respiratory specialists. This distance learning portal contains up-to-date study material for the state-of-the-art in Pulmonology.
Reign of Terroir was a wine blog with the theme "From the Vineyard to the Glass, Winemaking in an age of High Tech."
Senaste inom hälsa, biologi & virologi
The ECRD is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place.
PerkinElmer enables scientists, researchers and clinicians to address their most critical challenges across science and healthcare. We provide solutions for the diagnostics, life sciences, food and applied markets.