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Rare Diseases

Get a global overview and learn all about changing world of Rare diseases

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Science in Rare Diseases

Get a 360º view and stay updated on Rare diseases with content selected for you

Every Moment Matters: Navigating Diagnosis of Fabry Disease

Every Moment Matters: Navigating Diagnosis of Fabry Disease

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Uncover ASMD and GAUCHER

Uncover ASMD and GAUCHER

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Legacy Video for Rare Disease Academy

Legacy Video for Rare Disease Academy

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ASMD

ASMD

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What Are the Signs and Symptoms of ASMD?

What Are the Signs and Symptoms of ASMD?

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Gaucher

Gaucher

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Navigating the Pompe Disease Journey

Navigating the Pompe Disease Journey

Recognizing Key Signs and Symptoms Along the Way

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Better care for Fabry
  • Rare Diseases

Better care for Fabry

Every Moment Matters on the Fabry Journey

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Inspiration and innovation

Get inspired and stay at the forefront of innovation with the following resources

Better care for Fabry
  • Rare Diseases

Better care for Fabry

Every Moment Matters on the Fabry Journey

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How To Diagnose LSDs?
  • Rare Diseases

How To Diagnose LSDs?

Learn more about early diagnosis of lysosomal storage disorders: Fabry disease, Pompe Disease, Gaucher disease, Acid Sphingomyelinase Deficiency (ASMD, also known as Niemann-Pick Type A/B), mucopolysaccharidoses (MPSs).

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Pompe don’t lose time video
  • Rare Diseases

Pompe don’t lose time video

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Gaucher disease awareness month video
  • Rare Diseases

Gaucher disease awareness month video

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What are the new diagnostics modalities used to expedite Rare Disease patients identification?
  • Rare Diseases

What are the new diagnostics modalities used to expedite Rare Disease patients identification?

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Diagnostic Algorithm for Gaucher Disease in Pediatric
  • Rare Diseases

Diagnostic Algorithm for Gaucher Disease in Pediatric

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Diagnostic Algorithm for Gaucher Disease in Adult

Diagnostic Algorithm for Gaucher Disease in Adult

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Bone Monitoring Guidelines in Gaucher Disease Video
  • Rare Diseases

Bone Monitoring Guidelines in Gaucher Disease Video

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Events

Stay up to date and register for the latest events and view webinar recordings.

Do not Keep it masked! Prof. Fatih Ezgü

Do not Keep it masked! Prof. Fatih Ezgü

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Introduction to Gaucher Disease by Prof. Moeenaldeen D. Alsayed

Introduction to Gaucher Disease by Prof. Moeenaldeen D. Alsayed

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ASMD: Overview And Clinical Cases - by Prof. Roberto Giugliani

ASMD: Overview And Clinical Cases - by Prof. Roberto Giugliani

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Dr. Rana Felemban lecture on MPS disease

Dr. Rana Felemban lecture on MPS disease

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Prof. Christina Lampe lecture on MPSI disease

Prof. Christina Lampe lecture on MPSI disease

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