Ajay K. Nooka, MD, Associate Professor of Hematology and Medical Oncology at Emory School of Medicine, discusses the updated efficacy and safety results from the phase 1/2 MajesTEC-1 study evaluating teclistamab in patients with relapsed or refractory multiple myeloma.

Multiple myeloma is a rare blood cancer associated with uncontrolled growth of plasma cells. Abnormal plasma cells – also known as myeloma cells – interfere with the production of healthy blood cells in the bone marrow. Symptoms of multiple myeloma may include: bone pain (particularly in the chest and spine), frequent infections, weakness or numbness in the legs, fatigue, confusion, excessive thirst, and constipation. While the disease is treatable, relapses are common and some patients are refractory to first line, and subsequent, therapies.

This CME program highlights the latest clinical research about pulmonary arterial hypertension (PAH). PAH is a rare, progressive disorder characterized by high blood pressure in the pulmonary arteries.

Symptoms of PAH include shortness of breath (dyspnea) especially during exercise, chest pain, and fainting episodes. The progressive nature of this disease means that an individual may experience only mild symptoms at first, but will eventually require treatment and medical care to maintain a reasonable quality of life. There are numerous treatment options and options in development for persons with PAH and it is imperative clinicians who manage these patients stay up-to-date on the latest clinical research.

This CME program, hosted by Jean Elwing, MD, of the University of Cincinnati College of Medicine. It is supported by an educational grant from Merck.

To earn CME credit, go to https://checkrare.com/learning/p-pah-research-highlights-chest-2022/

Dr. Harrison Farber, a pulmonologist and director of the Pulmonary Embolism Response Team at Tufts Medical Center discusses a chronic rare disease that affects the circulatory system in the lungs and directly affects the ability of the lungs to function.

Pulmonary Arterial Hypertension, or PAH, is characterized by shortness of breath, dizziness, and chest pressure. Dr. Harrison Farber joins us to discuss the impact of COVID-19 on PAH patient care.

This CME/CE activity, hosted by Ozlem Goker-Alpan, MD, Co-founder and President Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) and guest lecturer, Oral Alpan, MD, of Amerimmune in McLean, VA, provides an overview of our current understanding of the immune system in the pathophysiology and management of lysosomal disorders.

At the end of this activity, participants should be able to:

• Describe how the immune system is involved in Lysosomal Storage Disorders.
• Review the pathophysiology of immune response reactions.
• Describe how to better manage patients with immune response reactions.
• Describe the best clinical practices to monitor the patients with immune response reactions.

Educational Support for this activity was provided by Takeda, Sanofi, and Chiesi.

To obtain CME credit, please visit https://checkrare.com/learning-center/courses/

This 15-minute CME program highlights the latest clinical research about Growth Hormone Deficiency (GHD) and is hosted by a leading expert in GHD, Paul Saenger, of the Albert Einstein College of Medicine.

GHD is a rare endocrine disorder characterized by insufficient levels of growth hormone being secreted from the anterior pituitary gland. A hallmark of prolonged GHD is growth retardation or deceleration, as well as short stature. Additionally, growth hormone deficiency is associated with metabolic abnormalities, impaired cardiovascular function, fatigue, delayed or incomplete puberty, osteoporosis, and reduced muscle strength. Given the varied clinical profile of these patients, GHD should be managed by a multidisciplinary team lead by a pediatric or adult endocrinologist. There are numerous treatment options and options in development for persons with GHD and it is imperative clinicians who manage these patients stay up-to-date on the latest clinical research.
Supported by an educational grant from Novo Nordisk, Inc.

To receive CME credit for this program, visit https://checkrare.com/learning-center/courses/

Guillaume Canaud, MD, PhD, of the Paris Descartes University, explains why it is important to regularly measure objective outcomes in persons with PiK3CA-related overgrowth syndrome (PROS).

PROS is a group of rare congenital disorders that lead to the overgrowth of parts of the body. PROS is caused by gain of function mutations in the PIK3CA gene. Specific disorders under the umbrella of PROS include fibroadipose hyperplasia, hemihyperplasia multiple lipomatosis (HHML), CLOVES syndrome, macrodactyly, fibroadipose infiltrating lipomatosis, megalencephaly-capillary malformation (MCAP), and dysplastic megalencephaly (DMEG).

Sean J. Pittock, MD, Director of Mayo Clinic's Center for Multiple Sclerosis and Autoimmune Neurology and of Mayo's Neuroimmunology Laboratory discusses the latest results from Phase III CHAMPION-NMOSD trial recently presented at European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress. The results showed that treatment with ravulizumab-cwvz significantly reduced relapse risk in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).

NMOSD is a rare central nervous disorder that primarily affects the spinal cord and optic nerves. Symptoms of NMOSD may include blindness in one or both eyes, weakness or paralysis of arms or legs, spasming, loss of sensation, uncontrollable vomiting and hiccups, and bladder/bowel problems due to spinal cord damage. Relapse is very common in persons with NMOSD and episodes can be severe enough to cause permanent disability.

As Dr. Pittock explains, the CHAMPION-NMOSD study is a global Phase III, open-label, multicenter trial evaluating the safety and efficacy of ravulizumab in adults with AQP4-Ab+ NMOSD (n-58). The data presented by Dr. Pittock at ECTRIMS showed that no relapses were observed in patients receiving ravulizumab, with a median treatment duration of 73 weeks. Further, 100% of patients receiving ravulizumab remained relapse-free at 48 weeks, compared to 63% of patients in the external placebo arm.

To learn more about NMOSD and other rare neurologic disorders, visit checkrare.com/ neurology/

Angela Scheuerle, MD, Medical Geneticist at the UT Southwestern Medical Center, discusses National Organization for Rare Disorders’ (NORD) Rare Disease Centers of Excellence of which UT Southwestern is one.

There are currently 31 NORD Rare Disease Centers of Excellence across the United States. The primary goal for establishing this network was to advance care and expand access for rare disease patients. The second goal was to enable rare disease experts to work collaboratively, which will hopefully lead to faster progress in terms of diagnoses, treatments, and development of guidelines and new therapies.

Many of the Centers of Excellence specialize in particular rare disease areas; for example, at UT Southwestern, they are best known for their work in lipid metabolism disorders and cholesterol diseases. Additionally, UT Southwestern can care for rare disease patients across the lifespan as they have rare disease programs for all ages and, for some rare diseases, have standardized programs to transition patients from youth- to adult-centered care.

This CME/CE activity, hosted by Ozlem Goker-Alpan, MD, Co-founder and President Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) and Sonata Jodele, MD, Research Professor of Pediatrics at Cincinnati Children’s Hospital Medical Center, highlights the current trends in gene therapy for lysosomal storage diseases as well as some of the safety concerns with such therapy.

At the end of this activity, participants should be able to:
Describe the limitations and unmet needs of the current therapeutic landscape for lysosomal disorders
Review the gene transfer therapies for lysosomal disorders
Describe the best clinical practices to monitor the safety profile of gene therapy
Describe the best practices for gene therapies

Educational Support for this activity was provided by Takeda, Sanofi, and Chiesi.

To obtain CME credit, please visit https://checkrare.com/learning-center/courses/

This CME/CE activity, hosted by Ozlem Goker-Alpan, MD, Co-founder and President Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) and Sonata Jodele, MD, Research Professor of Pediatrics at Cincinnati Children’s Hospital Medical Center, highlights the current trends in gene therapy for lysosomal storage diseases as well as some of the safety concerns with such therapy.

At the end of this activity, participants should be able to:
Describe the limitations and unmet needs of the current therapeutic landscape for lysosomal disorders:
- Review the gene transfer therapies for lysosomal disorders
- Describe the best clinical practices to monitor the safety profile of gene therapy
- Describe the best practices for gene therapies

Educational Support for this activity was provided by Takeda, Sanofi, and Chiesi.

To obtain CME credit, please visit https://checkrare.com/learning-center/courses/