SOCIETY FOR THE STUDY
OF INBORN ERRORS OF METABOLISM

The aim of the society is to foster the study of inherited
metabolic disorders and related topics.


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4 - 5 November 2021

Drug Development for Rare Diseases: Where are we now, how did we get here and where do we want to be?
An online course

Description: Orphan drug legislation has led to an explosion in the number of drugs being licensed for rare diseases, but has it been successful in its aim of making effective treatments accessible to patients affected by rare diseases? We will discuss challenges facing clinicians, regulatory agencies and manufacturers who are working to provide treatment to patients with rare diseases. We will use experience from the care of patients with lysosomal storage diseases to discuss orphan drug policies, real world evidence, and barriers to access. The course will have pre-recorded and live interactive sessions to develop dialogue on these challenging issues. 

Scientific Organising Committee: Sandra Sirrs, Vancouver and Robin Lachmann, London

Dates: 4th and 5th November between 2-7pm CET – virtual

See the course flyer here.

See the course programme here.

Register here.

See ssss

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    Drug Development for Rare Diseases: Where are we now, how did we get here and where do we want to be?
    An online course

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    14TH INTERNATIONAL CONGRESS OF INBORN ERRORS OF METABOLISM

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  • 1

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Society for the Study of Inborn
Errors of Metabolism (SSIEM)
PO Box 3375,
South Croydon, CR2 1PN 
United Kingdom

Registered Office: 
The Society for Study of Inborn Errors of Metabolism, C/O Stone King LLP,
Boundary House,
91 Charterhouse Street,
London, United Kingdom, EC1M 6HR

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SSIEM E-Learning

now released
HYPERAMMONAEMIA
Online learning resource

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