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Fabry Disease Highlights from WORLDSymposium 2020

At WORLDSymposium 2020, new information and data were presented on Fabry disease, a rare lysosomal storage disorder. This CME/CNE activity highlights the latest information about Fabry disease from WORLDSymposium and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patients with this rare disease.
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Gaucher Disease Highlights from WORLDSymposium 2020

At WORLDSymposium 2020, new information and data were presented on Gaucher disease, a rare lysosomal storage disorder. This CME/CNE activity highlights the latest information about Gaucher disease from WORLDSymposium and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patients with this rare disease.
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Managing Individuals With Eosinophilic Granulomatosis With Polyangiitis (EGPA)

The goal of this educational initiative is to improve the knowledge and ability of clinicians who may encounter individuals with EGPA to recognize, manage, and treat patients with EGPA. Unfortunately, many clinicians may encounter individuals who are living with undiagnosed Eosinophilic Granulomatosis with Polyangiitis (EGPA). Although these patients could gain benefit from treatment, damage ...
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Mucopolysaccharidoses (MPSs) Highlights from WORLDSymposium 2020

At WORLDSymposium 2020, new information and data were presented on Mucopolysaccharidoses (MPSs), a group of genetic lysosomal disorders. This CME activity highlights the latest information about MPSs from WORLDSymposium and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patients with these rare diseases.
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Mucopolysaccharidosis I

Early symptoms for Mucopolysaccharidosis I (MPS I), especially in those with the attenuated form of the disease, can be very difficult to distinguish from other more common conditions. Since this progressive disease has a treatment that can slow progression, it is imperative that clinicians recognize symptoms early so a correct diagnosis can be made. In this module, our faculty educator will exp...
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Neuroendocrine Tumors (NETs) Research Highlights from ENDO 2020

Although the 2020 Endocrine Society’s annual meeting (ENDO 2020) did not take place as scheduled, abstracts, including new information and data on neuroendocrine tumors (NETs) were published. This CME activity highlights the latest information about NETs from ENDO 2020 and provides expert analysis of its clinical relevance for busy members of the care team in order to help them care for patien...
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Rare Disease Clinical Trials

When caring for an individual with a rare disease who has exhausted standard treatments or for whom there are no treatments available, exploration of clinical trials, and possibly expanded access programs, may be warranted. However, many care team members and their patients may not know where to begin with these options. In addition, a number of barriers to participation may exist for both the p...
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Suspecting ATTR Amyloidosis

Amyloid transthyretin (ATTR) amyloidosis is a rare form of amyloidosis. The most dominant organ usually impacted by amyloidosis is the heart, but it can also impact other organs, including the nervous system. Many clinicians who see these patients before they are diagnosed will not be aware of ATTR amyoidosis, nor when to suspect it and how to diagnose this rare condition. On average it takes 2 ...
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TTR Amyloidosis

By learning more about the pathophysiology of rare diseases like TTR Amyloidosis, members of the care team who may encounter these patients on their diagnostic journeys can have a profound impact on their ultimate outcomes.
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Tumor-induced Osteomalacia (TIO): Diagnosis and Management

Tumor-induced osteomalacia (TIO) is a rare paraneoplastic condition commonly characterized by bone pain, muscle weakness and fractures. These tumors secrete excess fibroblast growth factor 23 (FGF23) which inhibits the sodium phosphate renal co-transporters and suppresses 1α hydroxylase activity, thereby decreasing renal reabsorption and increased urinary phosphate excretion. As a result, unexp...
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