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New and Emerging Phenotypes in Lysosomal Storage Diseases

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.
Expiration	Dec 15, 2022
Discipline(s)	Physician CME, Physician Assistant CME
Format	Online, Podcast, Webinar / Webcast / Video
Credits / Hours	1.0
Accreditation	ACCME
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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