Rare and Orphan Diseases

Guest: Mohamed Ibrahim, MB, BCh

IgA nephropathy (IgAN) is a rare autoimmune kidney disease in which IgA deposits damage kidney filters, causing symptoms like proteinuria and swelling. While symptoms can overlap with other kidney diseases, IgAN presents challenges of its own. Learn about the unique characteristics of IgAN with Dr. Mohamed Ibrahim, an Assistant Professor of Transplant Nephrology at the University of Maryland School of Medicine.

Guest: Jennifer Moore, MS, RDN, CSR, LDN

Host: Charles Turck, PharmD, BCPS, BCCCP

IgA nephropathy, or IgAN, is a rare kidney disease that requires dietary changes to maintain kidney health and function while supporting other organ systems. Learn about the most crucial dietary considerations for these patients from Jennifer Moore, a renal nutrition expert.

Host: Gates B. Colbert, MD

Guest: Kelly Helm

Over 35 million people in the United States suffer from some form of chronic kidney disease, but many of them lack the support and education to navigate their condition and receive the best possible care. After her daughter was diagnosed with a rare kidney disease, Kelly Helm discovered the many obstacles associated with management and treatment. Hear from Kelly as she sits down with Dr. Gates Colbert to share what she’s learned as a parent, caregiver, and advocate. Kelly is the Executive Director of Patient Engagement at NephCure, a nonprofit organization dedicated to supporting and empowering patients with rare kidney diseases.

Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Mary Frances McMullin, MD

The MOMENTUM study evaluated the impact of the treatment option momelotinib on patient-reported outcomes, including symptom burden and overall quality of life. Joining Dr. Charles Turck to share the key findings and potential implications for myelofibrosis care is Dr. Mary Francis McMullin, who co-authored and presented the poster at the American Society of Hematology (ASH) Annual Meeting and Exposition.

Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Douglas Tremblay, MD

Based on three randomized-controlled trials, the JAK inhibitor momelotinib has demonstrated clinical activity against anemia, constitutional symptoms, and splenomegaly in patients with myelofibrosis. But what do we know about its long-term safety? That’s what a recent study explored, and now, Dr. Douglas Trembley is here to share the findings from the largest clinical trial safety database for a JAK inhibitor in myelofibrosis. Dr. Trembley is an Assistant Professor at the Icahn School of Medicine at Mount Sinai in New York.

Guest: Kelly G. Gwathmey, MD

ALS can take up to a year to diagnose, but researchers are hard at work trying to find ways to minimize diagnostic delays and errors. One such solution is the Rapid Access ALS Clinic, where patients were diagnosed an average of two months sooner. Here to talk about this work is Dr. Kelly Gwathmey, Professor of Neurology at the Virginia Commonwealth University School of Medicine who presented a session on this topic at the 2024 American Association of Neuromuscular and Electrodiagnostic Medicine Annual Meeting.

Host: Gates B. Colbert, MD

Guest: Ryan Cassaday, MD

According to the available long-term data, CAR T-cell therapy boosts overall survival and durable remission rates in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL). Given this data, it’s important to know how we can best identify appropriate patients for this approach and manage adverse events so they can achieve those long-term benefits. To learn more about the available data and key considerations for using CAR T-cell therapy to treat relapsed/refractory B-ALL, Dr. Gates Colbert speaks with Dr. Ryan Cassaday, Associate Professor in the Division of Hematology-Oncology at the University of Washington School of Medicine in Seattle.

Host: Denis Hadjiliadis, MD, MHS, PhD

Guest: Gregory S. Sawicki, MD, MPH

Guest: Alexandra Wilson, MS, RDN, CDE

Guest: Susanna A. McColley, MD

Cystic Fibrosis (CF) care faces challenges from health disparities, limited access, and unintended bias, affecting patient outcomes. This CME program aims to equip healthcare professionals with the skills needed to overcome these barriers and provide equitable care for all CF patients. It covers telehealth strategies, best practices for transitioning patients from pediatric to adult care, and managing co-morbidities. The program also explores how evolving treatments impact patient outcomes.

Host: Amir T. Fathi, MD

Guest: Courtney DiNardo, MD

Guest: Brian A. Jonas, MD, PhD

While the expanding arsenal of therapies available for patients with AML allows practitioners to further hone treatment and individualize care, the complexity of treatment decision-making requires careful consideration of potential risks and benefits of each treatment option. This real-world, case-based program will provide a format for community hematology-oncologists to hear current updates on the latest targeted therapies in AML and engage in pointed, practical discussions on how to best integrate these data into current and local AML treatment paradigms.

Guest: Betsy O'Donnell, MD

Plasma cell disorders range from multiple myeloma to the monoclonal gammopathy of undetermined significance (MGUS), which is a benign condition that affects anywhere between 3 and 10 percent of the population starting at age 50. However, about 1 percent of people per year who have MGUS will progress to multiple myeloma. Here to talk about common precursor diseases like MGUS and smoldering myeloma and how they can progress to multiple myeloma is Dr. Elizabeth O’Donnell, Director of Early Detection and Prevention at Dana-Farber.