Rare and Orphan Diseases

Fibrodysplasia ossificans progressiva (FOP) is a severely disabling myopathy in which extraskeletal bone forms and accumulates over time within muscles and soft tissues.1,2 Here’s what we know so far about the mechanisms of heterotopic ossification in patients with FOP.

References:

1. Kaplan FS et al. Best Pract Res Clin Rheumatol 2008;22:191–205.
2. Pignolo RJ et al. Orphanet J Rare Dis 2011;6:80.

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Guest: Mateo Mejia Saldarriaga, M.D.

Mateo Mejia Saldarriaga, M.D., a hematologist/oncologist at NewYork-Presbyterian and Weill Cornell Medicine, explains how he and his team conducted a retrospective study that identified a biomarker to enhance treatment planning for BCMA CAR T-cell therapy in multiple myeloma. By measuring absolute lymphocyte count (ALC) through a routine CBC 15 days after a CAR T-cell therapy injection, they found patients with an ALC > 1,000 had a median progression-free survival (PFS) of 30 months whereas patients with an ALC ≤ 500 had a median PFS of 6 months. This new biomarker is now being leveraged to help doctors predict whether a patient is benefiting from treatment in as early as 15 days.

© 2025 NewYork-Presbyterian

Guest: Abiodun Ologunowa

Pediatric sickle cell disease treatment, particularly the use of hydroxyurea, NSAIDs, and opioids, has evolved in response to clinical guidelines and regulatory shifts, but gaps still remain in how children receive essential medications. Joining Dr. Charles Turck to discuss these national prescribing trends, disparities in care, and the implications of evolving treatment guidelines for this population is Dr. Abiodun Ologunowa. Dr. Ologunowa is a doctoral candidate and research assistant in the Department of Pharmacy Practice and Clinical Research at the University of Rhode Island College of Pharmacy.

Guest: Yogindra Persuad

Despite the promise of curative therapies for sickle cell disease, access and cost barriers highlight the ongoing need for disease-modifying treatments like hydroxyurea. Tune in to hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children’s Research Hospital in Memphis, Tennessee, discuss a multimodal approach to care, emphasizing research on oral agents and broader symptom management beyond pain.

Guest: Nidhi Bhatt

Effective transition from pediatric to adult care should begin as early as age twelve for patients with sickle cell disease, incorporating factors like education, self-advocacy skills, and support from transition care coordinators. Tune in to hear Dr. Nidhi Bhatt, who works in the Department of Hematology at St. Jude Children’s Hospital in Memphis, Tennessee, discuss how involving patients and families early on helps identify barriers and improve long-term outcomes.

Guest: Parul Rai

Hydroxyurea and chronic transfusions remain cornerstone therapies in managing sickle cell disease, with proven benefits in reducing stroke risk and improving anemia. However, considerations remain, including their impact on cardiac health. Learn about these long-standing therapies and best practices for using them to manage sickle cell disease with Dr. Parul Rai. Dr. Rai is a physician in the Department of Hematology at St. Jude’s Hospital in Memphis, Tennessee.

Guest: Parul Rai

Cardiopulmonary complications are a leading cause of early mortality in adults with sickle cell disease, with evidence showing that cardiac injury may begin as early as childhood. However, despite the severity of these issues, there are currently no uniform guidelines for asymptomatic cardiac screening in this population. Join Dr. Parul Rai, a physician in the Department of Hematology at St. Jude’s Hospital in Memphis, Tennessee, to learn about current research, early detection strategies, and the need for more sensitive diagnostic markers to prevent severe cardiac outcomes in patients with sickle cell disease.

Guest: Yogindra Persuad

For decades, hydroxyurea has been the cornerstone therapy for sickle cell disease, but new disease-modifying therapies, curative strategies, and gene therapies are expanding options for patients. While these advancing approaches come with some risks, they can help offer symptom relief, reduce complications, and give patients more choices when it comes to managing their sickle cell disease. Hear Dr. Yogindra Persuad, a physician in the Department of Hematology at St. Jude Children’s Research Hospital in Memphis, Tennessee, walk through the history of sickle cell disease treatment and discuss the benefits and risks of these developing therapies.

Host: Crystal Proud, MD

Guest: Erika Finanger, MD, MS

By completing this activity, learners will gain the skills and confidence to determine therapeutic candidacy and conduct appropriate assessments for patients with Duchenne Muscular Dystrophy (DMD) across the age spectrum, including older and non-ambulatory individuals. They will improve their ability to interpret and apply the latest clinical data to real-world scenarios and make informed decisions about treatment and monitoring. Through case-based education, learners will enhance their competence in aligning patients to appropriate therapies and managing long-term care in a rapidly evolving treatment landscape.

Guest: Nicolas Dubuisson, MD

Host: Ryan Quigley

Early recognition of CIDP is essential to prevent irreversible damage and guide appropriate treatment, but its varied presentations can complicate diagnosis. Ryan Quigley sits down with Dr. Nicolas Dubuisson to discuss the clinical hallmarks of CIDP, its variants, and key risk factors. Dr. Dubuisson is a Postdoctoral Researcher at the Nuffield Department of Clinical Neurosciences at Oxford University in England.