Maladies métaboliques héréditaires

ci dessous une des maladies métaboliques héréditaires
Niemann Pick
due à un dysfonctionnement du métabolisme cellulaire d’origine génétique

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– Pinpoint opportunities to fast-track evidence-based treatments to patients
– Gather strategies for identifying patient-centered clinical endpoints
– Learn about success stories involving pharma
– Learn about patient partnerships and accelerated clinical trials
– Uncover strategies to involve the patient early and often throughout the life cycle of drug development and implementation
Benefit from updates on the regulatory guidelines determining the clinical and market access development potential for orphan drugs

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