Science in Rare Diseases
Get a 360º view and stay updated on Rare diseases with content selected for you
Gaucher
MPSI
Pompe
Fabry
What is ASMD?
MPSI – Rheumatology Case Study
How is ASMD inherited?
MPSI – ENT Case Study
MPSI – Endocrinology Case Study
Pompe don’t lose time video
MPSI chase the signs video
Gaucher disease awareness month video
Inspiration and innovation
Get inspired and stay at the forefront of innovation with the following resources
Gaucher
MPSI
Pompe
Fabry
IOPD Challenges
LSDs overview
ASMD PhenoTypes
Diagnostic challenges of LOPD
MPSI – ENT Case Study
The Stigma of Rare Diseases
Pompe don’t lose time video
MPSI chase the signs video
Events
Stay up to date and register for the latest events and view webinar recordings.
Introduction to Gaucher Disease by Prof. Moeenaldeen D. Alsayed
Do not Keep it masked! Prof. Fatih Ezgü
Prof. Christina Lampe lecture on MPSI disease
Dr. Rana Felemban lecture on MPS disease
