In this episode, we discuss a new formulation of treatment for HAE, the FDA approval of Voceryq for recurrent respiratory papillomatosis, and 3D printed treatment options.

Produced by CheckRare.

Explore additional CME activities, physician interviews, podcasts, and rare disease resources at CheckRare.com. Subscribe to the CheckRare Podcast Network for the latest rare disease education, expert interviews, and weekly news.

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

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In this episode, we discuss a new formulation of treatment for HAE, the FDA approval of Voceryq for recurrent respiratory papillomatosis, and 3D printed treatment options.

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Sarah Cheng, MD, PhD, a clinical oculoplastic fellow at the University of California, San Diego, discusses findings from an exploratory study examining the biological changes in orbital tissue associated with Tepezza  (teprotumumab-trbw) treatment. The findings were presented at the 2026 American Academy of Ophthalmology (AAO) Annual Meeting in New Orleans, Louisiana.



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.

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Maria Picone, co-founder and CEO of the TREND Community, and mother of a 14-year-old with Prader-Willi syndrome (PWS), discusses her family’s journey with PWS, including why more research and better data are so important.

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Mirum Pharmaceuticals recently announced positive topline results from its phase 3 EXPAND study, evaluating Livmarli (maralixibat) for cholestatic pruritus in patients six months and older with ultra-rare cholestatic liver diseases. Jennifer Vittorio, MD, an EXPAND investigator, an Associate Professor of Pediatrics, and the Medical Director of Pediatric Liver Transplantation at NYU Langone Health, discusses the findings. 

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.

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What were the results from the EXPAND clinical trial of maralixibat in patients with ultra-rare cholestatic liver diseases?

View the full interview at https://checkrare.com/mirums-livmarli-achieves-primary-endpoint-in-phase-3-expand-study/

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Sarah Cheng, MD, PhD, a clinical oculoplastic fellow at the University of California, San Diego, discusses findings from an exploratory study examining the biological changes in orbital tissue associated with Tepezza (teprotumumab-trbw) treatment. The findings were presented at the 2026 American Academy of Ophthalmology (AAO) Annual Meeting in New Orleans, Louisiana.

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View the full interview at https://checkrare.com/mirums-livmarli-achieves-primary-endpoint-in-phase-3-expand-study/

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Nisha Joseph, MD, Associate Professor in the Department of Hematology and Medical Oncology at the Emory University School of Medicine, discusses how bispecific antibodies are changing multiple myeloma treatment. 

Multiple myeloma is a rare cancer of the plasma cells and the most common type of plasma tumor. Plasma cells are white blood cells that make antibodies to protect against infection. In multiple myeloma, these cells multiply rapidly, crowding out normal cells in the bone marrow and affecting the production of red blood cells, platelets, and other white blood cells. Symptoms include bones that break easily, frequent infections, fatigue, bruising, and bleeding. About 36,000 Americans are diagnosed with multiple myeloma each year.

Multiple myeloma is challenging to treat. Between 30% and 50% of patients don’t respond to first-line therapy, and almost everyone who initially achieves remission relapses or develops resistance over time. Because of this, researchers are always looking for new ways to combat the disease. 

Dr. Joseph said that bispecific antibodies are especially promising because they help a patient’s T cells recognize and attack the cancer. She described the process in simple terms. “It basically gloms onto the T cell, gloms onto the myeloma cell, and says, ‘Hey, look over here. Kill this guy.’” 

Furthermore, Dr. Joseph shared that bispecific antibodies can be combined with other myeloma drugs because they fight cancer in different ways. For example, some drugs, such as immunomodulatory agents and cereblon E3 ligase modulatory drugs (CELMoDs), stimulate the immune cells, while bispecific antibodies direct T cells to attack myeloma directly. Researchers are trying to determine whether combining these treatments can improve patient outcomes. 

Despite their potential benefits, Dr. Joseph said that undergoing bispecific antibody treatment requires short-term and long-term considerations. 

In the short term, bispecific antibodies are typically administered via a ramp-up period that takes about a week. Initial doses are small, but they increase over time, and that process requires several doctor visits. Dr. Joseph shared that this approach reduces the risk of cytokine release syndrome (CRS), which can cause fever, low blood pressure, and shortness of breath. Some patients may also experience neurological symptoms, which can require caregiver support.

The good news is that after the initial ramp-up period, Dr. Joseph said these side effects generally become much less of a concern.  

In the long term, bispecific antibodies can increase the risk of infection and low blood counts. Dr. Joseph noted that clinicians have learned that when it comes to using these drugs, “less is more.” She then explained that once a patient responds to treatment, doctors typically extend the time between doses to make it easier on the immune system. Preventive measures, such as antibody infusions and antibiotics, can also support these efforts.

Next, Dr. Joseph emphasized the importance of putting patients first when using bispecific antibodies. Even though these drugs are effective and typically well-tolerated, treatment is an ongoing process, requiring routine visits, something she described as “a marathon, not a sprint.” 

Even so, these drugs make multiple myeloma treatment more accessible. Unlike CAR T-cell therapy, which is primarily offered at larger academic centers with the necessary infrastructure, bispecific antibodies can be administered at smaller facilities, like community treatment centers. 

To learn more about multiple myeloma and other rare hematologic disorders, visit: https://checkrare.com/diseases/hematologic-disorders/

To learn more about Dr. Joseph’s background and areas of expertise, visit: https://winshipcancer.emory.edu/profiles/joseph-nisha.php

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.

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