Rare Discussions

This 30-min CME program provides an overview of the latest clinical research presented at ENDO 2023 involving Cushing’s disease.

Faculty
Lisa Nachtigall, MD
Clinical Director, Neuroendocrine & Pituitary Tumor Clinical Center
Massachusetts General Hospital
Associate Professor of Medicine
Harvard Medical School

Learning Objectives
- After participating in the activity, learners should be better able to:
- Describe the latest research being presented to better manage individuals with Cushing’s disease and its clinical relevance.
- Share new information with their clinical team.

Supported by an educational grant from Recordati Rare Diseases, Inc.

To obtain CME credit, go to https://checkrare.com/learning/p-cushings-disease-research-highlights-endo-2023/

Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.

Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

This CME/CE activity describes the pathophysiologies and management options for lysosomal disease patients with cardiomyopathies. This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE.

This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees. To receive credit for this program, go to https://checkrare.com/learning/p-ldrtc2022-webinar2-managing-cardiomyopathies-in-lysosomal-disorders/

Speakers
Ozlem Goker-Alpan, MD, Founder and President, LDRTC
John Jefferies, MD, Governor, American College Cardiology, Tennessee Chapter President, American Heart Association, Mid-South Chapter Research Member, St. Jude Children’s Research Hospital Team Cardiologist, Memphis Grizzlies


.Learning ObjectivesAt the end of this activity, participants should be able to:

• Describe the role of the cardiologist in the team approach to care
• Describe best practices to monitor cardiac symptoms in lysosomal disorders
• Describe best practices to treat cardiac symptoms in lysosomal disorders

Support for this educational activity was provided by Takeda, Sanofi, Amicus Therapeutics and Chiesi USA.




Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.

Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

This 30-minute CME program highlights the latest clinical research about acromegaly, a rare, endocrine disorder.

Activity Faculty Wenyu Huang, MD, PhD Associate Professor Northwestern University Feinberg School of Medicine Chicago, IL

Support for this accredited continuing education activity has been made possible through educational grants from Recordati Rare Diseases Inc. and Ipsen Biopharmaceuticals, Inc.
To earn a CME credit, go to https://checkrare.com/learning/p-acromegaly-research-highlights-endo-2023/

Estimated time to complete: 0.5 hours Start date: Sep 30, 2023 End date: September 29, 2024

Target Audience: This activity has been designed to meet the educational needs of physicians specializing in endocrinology, neurosurgery, and family practice who may care for individuals with acromegaly. Other healthcare providers may also participate. Learning
Objectives After participating in the activity, learners should be better able to • Describe the latest research being presented to better manage individuals with acromegaly and its clinical relevance.

Accreditation and Credit Designation In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team. Physicians American Academy of CME, Inc., designates this enduring material for a maximum of 0.5 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPs: Other members of the care team will receive a certificate of participation.

Disclosure Statement According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated.

Disclosure of relevant financial relationships are as follows: Dr. Huang discloses the following relevant financial relationships with ineligible companies to disclose: Contracted research: Amryt Pharma, CinCor Pharma, Corcept Therapeutics, Crinetics Pharmaceuticals, Ionis Pharmaceuticals, Ascendis Pharma, Spruce Bioscience Consulting: Novo Nordisk, Crinetics Pharmaceuticals, Spruce Bioscience o Consulting and Speaking: Amryt Pharma, Recordati Rare Diseases

Other planners for this activity have no relevant financial relationships with any ineligible companies. This activity will review off-label or investigational information.
The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE.
This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.
Method of Participation There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity.

To receive CME credit for your participation, please complete the pre and post-program assessments. Your certificate will be emailed to you in within 30 days.
Privacy For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm

For more information about CheckRare’s privacy policy, please access https://checkrare.com/privacy/

Contact For any questions, please contact: [email protected] Copyright © 2023.

This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).



Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.

Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

Raymond Douglas, MD, PhD, a world-leading clinician and thought leader in thyroid eye disease (TED) who has been integral to developing therapeutics for the disease, provides an overview of TED, including diagnosis challenges and current and emerging treatments for this rare disease.

TED is a rare autoimmune disease that can dramatically impact a person’s vision. The condition often occurs in people with hyperthyroidism or Graves’ disease. Common symptoms can include upper eyelid retraction, dry eyes, inflammation, light sensitivity, as well as the sensation of a foreign body present in the eye.

TED is most often associated with Graves’ disease (GD), but also can occur in association with hypothyroidism, euthyroidism, and Hashimoto’s thyroiditis. GD affects approximately 1% to 2% of the adult population, with an estimated 40% of GD patients subsequently developing TED over the course of their lifetime. The onset of TED typically occurs between 30 and 50 years of age, with the disease course more severe after age 50.

Dr. Douglas has been appointed as Chief Scientific Officer at Sling Therapeutics.

Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.

Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

This 30-minute CME program highlights the latest clinical research about myasthenia gravis, a rare, autoimmune disease that targets the neuromuscular junction.

Treatment of myasthenia gravis is highly individualized and depends greatly on the myasthenia gravis subtype of each patient as well as each patient’s comorbidities. There are currently four drugs approved by the FDA, eculizumab, efgartigimod, ravulizumab, and rozanolixizumab. There are also treatments in development. Clinical trial data on these therapies were presented at the American Academy of Neurology Annual Meeting (AAN 2023) held in Boston, MA.

This CME program, hosted by Vera Bril, MD, of the University Hospital Network in Toronto, Canada, provides an overview of the latest clinical research presented at AAN 2023 focused on myasthenia gravis.

Supported by educational grants from argenx US, Inc. and UCB Inc. For complete activity information and to obtain CME credit, please, go to https://checkrare.com/learning/p-myasthenia-gravis-research-highlights-aan-2023/



Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.

Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

This continuing education activity is provided through collaboration between the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), CheckRare CE, and AffinityCE. This activity provides continuing education credit for physicians, physician assistants, nurses, nurse practitioners, and genetic counselors. A statement of participation is available to other attendees.

Speakers

• Ozlem Goker-Alpan, MD Founder and President, Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)
• Al-Hertani, MD, Director of the BCH Metabolism and Lysosomal Programs, Boston Children’s Hospita; lAssociate Professor of Pediatrics, Harvard Medical School

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

• Describe the need for a team approach to care
• Describe best practices to build a multidisciplinary team for a new patient
• Describe best practices to maintain a multidisciplinary team

Support for this educational activity was provided by Takeda, Sanofi, Amicus Therapeutics, and Chiesi USA.

To earn credit for this CME, go to https://checkrare.com/learning/p-building-and-maintaining-a-multidisciplinary-team-for-lysosomal-disorders/



Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.

Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

Sindhu Ramchandren, MD, Global Clinical Leader at Janssen Pharmaceuticals, explains the pathophysiology of myasthenia gravis and the need for more targeted therapies.

Myasthenia gravis is an autoimmune, neuromuscular disorder characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eyes, eyelids, facial expressions, chewing, talking, and swallowing. The presence of antibodies against acetylcholine receptors in the neuromuscular junction usually causes the condition.

As Dr. Ramchandren explains, myasthenia gravis is an autoimmune disease in which the person’s own immune system attacks ACh-receptors on the neuromuscular junction. The current standard of care for myasthenia gravis is to suppress the immune system, usually with broad-acting immunosuppressants. Dr. Ramchandren believes a more targeted approach would be of greater benefit to patients. Janssen is currently developing nipocalimab, a monoclonal antibody that binds to neonatal Fc receptors (FcRn). FcRn is a protein that naturally drives IgG recycling in the body. By blocking FcRn, nipocalimab reduces the levels of IgG autoantibodies while preserving the rest of the immune system.

Recently, in a phase 2 clinical trial, patients with myasthenia gravis were given nipocalimab. As a result, a dose-dependent rapid effect was observed on various disease biomarkers, including reduced titers of autoantibodies. Currently, A phase 3 clinical trial is underway (NCT04951622). To learn more about this and other autoimmune disorders, go to checkrare.com/diseases/autoimmune-auto-inflammatory-disorders/



Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.

Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

Sindhu Ramchandren, MD, Global Clinical Leader at Janssen Pharmaceuticals, explains the pathophysiology of myasthenia gravis and the need for more targeted therapies.

Myasthenia gravis is an autoimmune, neuromuscular disorder characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eyes, eyelids, facial expressions, chewing, talking, and swallowing. The condition is usually caused by antibodies against acetylcholine receptors in the neuromuscular junction.

As Dr. Ramchandren explains, myasthenia gravis is an autoimmune disease in which the person’s own immune system attacks ACh-receptors on the neuromuscular junction. The current standard of care for myasthenia gravis is to suppress the immune system, usually with broad-acting immunosuppressants. Dr. Ramchandren believes a more targeted approach would be of greater benefit to patients. Janssen is currently developing nipocalimab, a monoclonal antibody that binds to neonatal Fc receptors (FcRn). FcRn are proteins that naturally drive IgG recycling in the body. By blocking FcRn, unplumb reduces the levels of IgG autoantibodies while preserving the rest of the immune system.

Recently, in a phase 2 clinical trial, patients with myasthenia gravis were given nipocalimab. As a result, a dose-dependent rapid effect was observed on various disease biomarkers, including reduced titers of autoantibodies.

Currently, a phase 3 clinical trial is underway (NCT04951622). To learn more about this and other autoimmune disorders, go to checkrare.com/diseases/autoimmune-auto-inflammatory-disorders/

Warm autoimmune hemolytic anemia (wAIHA) is the most common type (60-70%) of autoimmune hemolytic anemia (AIHA). In most cases, wAIHA is due an immunoglobulin G (IgG) autoantibody that binds to red blood cells (RBC), leading to hemolysis. Current recommendations for managing people with wAIHA are largely based on case series and retrospective studies involving off-label medications. Also, while there are currently no medications specifically approved to treat wAIHA, data are emerging on new therapies under investigation which may impact treatment in the future.

This 60-minute CME program, hosted by Irina Murakhovskaya, MD, of the Montefiore Medical Center, Albert Einstein College of Medicine, in New York, NY and Bruno Fattizzo, MD, of the University of Milan and Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, in Milan, Italy, describes current treatment options, and treatments in development, for patients with wAIHA.

Supported by an educational grant from Janssen Biotech.

For complete activity information and to obtain CME credit, please, go to



Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.

Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.

Warm autoimmune hemolytic anemia (wAIHA) is the most common type (60-70%) of autoimmune hemolytic anemia (AIHA). In most cases, wAIHA is due an immunoglobulin G (IgG) autoantibody that binds to red blood cells (RBC), leading to hemolysis.

Current recommendations for managing people with wAIHA are largely based on case series and retrospective studies involving off-label medications. Also, while there are currently no medications specifically approved to treat wAIHA, data are emerging on new therapies under investigation which may impact treatment in the future.

This 60-minute CME program, hosted by Irina Murakhovskaya, MD, of the Montefiore Medical Center, Albert Einstein College of Medicine, in New York, NY and Bruno Fattizzo, MD, of the University of Milan and Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, in Milan, Italy, describes common complications that can impact the management of patients with wAIHA.

Supported by an educational grant from Janssen Biotech.

For complete activity information and to obtain CME credit, please, go to www.checkrare.com



Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.

Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.

Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.

Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.