European Conference on Rare Diseases - Lisbon November 27 - 28th 2007
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Resume
This page contains summaries of the presentation for the category "New treatment and orphan drug development" as well as links to the documents.
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Dr Kerstin Westermark (Europe)
Seven years of orphan drugs policy: what’s next ?
Dr Anne-Marie Masquelier (France)
Preparing the European scenario for advanced therapies (gene therapy, cell therapy, future EU regulation)
Dr François Meyer (France)
Timely and equitable access to orphan medicines across Member States - The european HAS Workshop
Camelia Al-Khzouz, Paula Grigorescu-Sido (Romania)
Access to the diagnosis and treatment of patients with mucopolysaccharidosis type I in Romania
Summary: Work hypothesis: Mucopolysaccharidosis type I (MPZ type I, Hurler disease) is a monogenic autosomal recessive disease induced by a deficiency of the enzyme α-L-iduronidase, whose main characteristics are craniofacial dysmorphism, severe somatic retardation, organomegaly, progressive mental retardation that can develop into dementia, osteoarthropathy with flexion contracture, and corneal opacities.
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