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New and Emerging Phenotypes in Lysosomal Storage Diseases

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Overview / Abstract:

This CME activity highlights how modern treatment options for lysosomal storage disorders have created new phenotypes for these once terminal conditions. Understanding the ‘new normal’ for these conditions can help health care providers manage their patients more effectively.


Dec 15, 2022


Physician CME, Physician Assistant CME


Online, Podcast, Webinar / Webcast / Video

Credits / Hours




Presenters / Authors / Faculty

Dr. Ozlem Goker-Alpan
Founder and President
Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)

Dr. Uma Ramaswami
Consultant in Inherited Metabolic Disorders
Royal Free London Hospital

Activity Specialities / Related Topics

Counseling, Diagnosis / Assessment / Patient History, Genetics, Hematology, Internal Medicine, Neurology, Orthopedics, Pediatrics, Primary Care

Sponsors / Supporters / Grant Providers

Takeda, Cheisi, Ultragenyx Pharmaceutical, and Spark Therapeutics.

Keywords / Search Terms

CheckRare Lysosomal storage diseases, rare disease, gaucher, Fabry, Pompe, MPS, mucopolysaccharidoses, sly syndrome, phenotypes, lysosomal, enzyme replacement therapy, gene therapy, Dr. Uma Ramaswami, Dr. Ozlem Goker-Alpan, orphan drug, WORLDSymposium Free CE CME

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