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Exploring Novel Treatments for Rett Syndrome
STATEMENT OF NEED
Rett syndrome is a rare, debilitating neurodevelopmental disorder almost always associated with a spontaneous mutation in the methyl-CpG-binding protein 2 (MECP2) gene on the X-chromosome. Affected individuals experience loss of purposeful hand skills, abnormalities in gait, loss of spoken language, and stereotypic hand movements, with more severe manifestations including seizures, autistic features, autonomic nervous system dysfunction, breathing abnormalities, sleep dist... |
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Duchenne Muscular Dystrophy: Recognizing Its Earliest Signs in Infancy and Care Transition into Adulthood
Target Audience
The intended audience for this activity are neurologists, neuromuscular specialists, pediatric neurologists, genetic counselors, and pediatricians.
Program Overview
Join a panel of expert clinicians to identify the subtle and very early signs of DMD that may be detected before functional symptoms are noticed and that can help earlier diagnoses and appropriate interventions. Panelists will review the importance of matching mutations to therapeutic decisions, discuss emergin... |
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Diagnosis and Management of Patients With Predominant Skin Symptoms
The educational design of this activity addresses the needs of allergy/clinical immunology, gastroenterology, dermatology, and hematology clinicians who manage patients with nonadvanced systemic mastocytosis.
Nonadvanced systemic mastocytosis (SM) is a group of rare disorders characterized by mast cell accumulation that results in a significant and varied symptomatic burden to the patient. The most prevalent of these symptoms include gastrointestinal symptoms, dermatological symptoms, and ... |
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Making the Call in Nonadvanced Systemic Mastocytosis Optimizing Multidisciplinary Management
Series Overview
Nonadvanced systemic mastocytosis (SM) is a group of rare disorders characterized by mast cell accumulation that results in a significant and varied symptomatic burden to the patient. The most prevalent of these symptoms include gastrointestinal symptoms, dermatological symptoms, and recurrent anaphylaxis in response to a variety of triggers such as food, insect venom, and medications. In this Phone-a-Friend series, a multidisciplinary panel of faculty experts will address the... |
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Hitting The Bullseye in NSCLC
Lung cancer is the second most common cancer in both men and women and is the leading cause of cancer-related deaths. The majority of lung cancer cases are categorized as NSCLC, which has a low overall 5-year survival rate, especially when it is diagnosed at advanced, unresectable stages. Recent advances in the treatment landscape have yielded promising improvements in progression-free survival and overall survival; however, outcomes are dependent on numerous factors, including the evaluation ... |
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LET'S TALK: Tackling Complex Issues Regarding SM-AHN Management A Monday Morning Joe Online Educational Activity
Systemic mastocytosis (SM) is a group of rare disorders characterized by the accumulation of mast cells in internal tissues and organs. The pathogenesis of SM is linked to a characteristic exon 17 D816V mutation of the KIT gene. Systemic mastocytosis with associated hematologic neoplasms (SM-AHN) is an advanced subtype of SM that is associated with a shortened median survival time. Recent advances in the SM therapeutic landscape have led to the development of KIT-targeted treatment options; ho... |
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Therapeutic Advancements for the Treatment of High-Risk MDS: Future Opportunities to Improve Patient Survival and Quality of Life
Stay up to date on the evolving risk classification for myelodysplastic syndromes (MDS) and current treatment guidelines for patients with high-risk disease in this self-directed education with leading oncology experts. Expert commentary from Courtney DiNardo, MD from MD Anderson Cancer Center and Dr. Dan Pollyea from the University of Colorado School of Medicine provides insights on emerging therapies that could soon improve management of high-risk MDS. Be ready to incorporate these emerging ... |
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Case Studies in HIV Resistance Testing and Treatment " Experienced Patients
Educational Need:
Through a series of patient cases, participants will explore the complexities of managing HIV infection in patients who have been treated with multiple different HIV regimens. Participants will engage in real-life case studies that will provide valuable insight into the intricacies of HIV treatment and resistance testing. Our faculty educators will discuss commonly encountered challenges faced by the care team and their treatment-experienced patients with HIV and offer real... |
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