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Gaucher Disease Research Highlights From the WORLDSymposium

This accredited CME activity, led by Gregory Grabowski, MD, Professor Emeritus at University of Cincinnati College of Medicine, highlights the latest research about Gaucher disease presented at WORLDSymposium 2022 along with expert analysis of its clinical relevance for busy members of the care team to help them care for patients they may encounter with this rare condition. Gaucher disease is...
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Hereditary Angioedema (HAE) Highlights from ACAAI 2021 Annual Meeting

This accredited CME activity provides a summary of the latest information about hereditary angioedema (HAE) that was presented at the American College of Allergy, Asthma, & immunology 2021 Annual Scientific Meeting (ACAAI 2021). Since the Covid-19 pandemic limited the ability for specialists to attend ACAAI 2021 and discuss this latest research, this program provides an expert review of the most...
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Lysosomal Storage Diseases: Central Symptoms and Comorbidities

This CME activity highlights how symptoms and comorbidities involving the central nervous system have a key role in how physicians manage patients with lysosomal storage disorders. Key medical problems discussed include cognitive deterioration, behavioral disorders, ophthalmologic problems, neuromuscular deterioration, Parkinson’s disease, depression, and seizures. Newer therapies that allow t...
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MPS II Research Highlights From the WORLDSymposium (Mucopolysaccharidosis type II; MPS II; Hunter syndrome)

This accredited CME activity, led by Barbara Burton, MD, Professor of Pediatrics at Northwestern University Feinberg School of Medicine, highlights the latest research about Mucopolysaccharidosis type II (MPS II; Hunter syndrome) presented at WORLDSymposium 2022 and provides expert analysis of its clinical relevance for busy members of the care team to help them care for patients they may encoun...
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New and Emerging Phenotypes in Lysosomal Storage Diseases

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.
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Newborn Screening: From RUSP to Reality

This is the first module in a four-part curriculum focused on Best Practices for Explaining Newborn Screening Results to Parents. This module will provide clinicians with the fundamentals of newborn screening.
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Newborn Screening: Hemoglobinopathies and Newer Disorders on the RUSP

This is the fourth module in a four-part curriculum focused on Best Practices for Explaining Newborn Screening Results to Parents. This module will focus on hemoglobinopathies and newer disorders which are part of the Recommended Uniform Screening Panel (RUSP) in order to better prepare clinicians to discuss positive results with new parents.
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Newborn Screening: Metabolic Conditions

This is the third module in a four-part curriculum focused on Best Practices for Explaining Newborn Screening Results to Parents. This module will focus on metabolic diseases which are part of the Recommended Uniform Screening Panel (RUSP) in order to better prepare clinicians to discuss positive results with new parents.
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Newborn Screening: Talking to Parents

This is the second module in a four-part curriculum focused on Best Practices for Explaining Newborn Screening Results to Parents. This module will focus on how to talk to parents about positive NBS results.
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Paroxysmal Nocturnal Hemoglobinuria (PNH) Highlights From ASH

At ASH, new information and data were presented on paroxysmal nocturnal hemoglobinuria (PNH)—a rare, acquired blood disease characterized by hemolytic anemia, bone marrow failure, thrombosis, and fatigue. This accredited CME activity highlights the latest information about PNH from ASH and provides expert analysis of its clinical relevance for busy members of the care team in order to help ...
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Research Highlights on Pyruvate Kinase (PK) Deficiency

This accredited CME activity provides a summary of the latest information about Pyruvate kinase (PK) deficiency that was presented at the 2021 American Society of Hematology Annual Meeting (ASH 2021). Since the Covid-19 pandemic limited the ability for specialists to attend ASH 2021 and discuss this latest research, this program provides a review by Rachael Grace, MD, of the most clinically-rele...
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