Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kimberly Ku, MD, an oncologist with Illinois Cancer Center and host of the podcast "Val-You Based Care,"

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Christine Clemson, PhD, senior vice-president and global head of medical affairs at Entrada Therapeutics, about exon skipping therapies in Duchenne.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Avril Daly, president of Paris-based Eurordis-Rare Diseases Europe, on how the EU is falling behind in clinical trials for rare diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jim Palma, CEO of the TargetCancer Foundation, a nonprofit organization that focuses on finding treatments for rare cancers.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Duchenne expert Paul Esteso, MD, PhD, medical director of the cardiac antithrombosis management program at Boston Children's Hospital.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Teresa Barnes, founder of PF Warriors—a global support network that provides education, inspiration and hope to individuals and families affected by pulmonary fibrosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Martina Cornel, MD, PhD, about the importance of adopting standardized newborn screening for rare diseases throughout Europe.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Diane Zapursky Quale, who founded the Bladder Cancer Advocacy Network 20 years ago with her late husband after he developed bladder cancer.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Laurah Turner, PhD, associate dean for artificial intelligence and educational informatics at the University of Cincinnati College of Medicine.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John MacMahon, a New Hampshire-based medical devices company that specializes in phototherapy to treat fatigue in people with multiple sclerosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Eric Small, MD, who recently stepped down as president of the American Society of Clinical Oncology (ASCO).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jessie Dubief, social research director of Eurordis, about the organization's new survey on the importance of addressing mental health among both patients and caregivers affected by rare diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Naomi Aziz, patient advocacy and engagement lead at the Endometrial Cancer Research Foundation. June is Uterine Cancer Awareness Month, and endometrial cancer now ranks as the nation's most common gynecologic cancer.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michelle Ng Gong, MD, president of the American Thoracic Society (ATS). Dr. Gong formally replaced Raed Dweik, MD, at the ATS 2026 conference in Orlando, Florida.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Andrew Wilson, MD, scientific director of the Alpha-1 Foundation, about the latest therapies to treat alpha-1 antitrypsin deficiency, an incurable genetic disease that causes both lung and liver damage.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Vallerie McLoughlin, MD, director of the Pulmonary Hypertension Program at the University of Michigan-Ann Arbor, about results of a phase 3 trial for ralinepag to treat pulmonary arterial hypertension.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Craig Martin, founder and CEO of the Orphan Therapeutics Accelerator (OTXL) — a nonprofit biotech dedicated to advancing shelved clinical-stage therapies for ultrarare diseases.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Henriette Farkas, MD, PhD, director of the Hungarian Angioedema Reference Center in Budapest, and a world expert on the treatment of HAE.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Antón Blatnik, PhD, who recently won a $150,000 grant from Cure SMA to study the molecular mechanisms that drive neuromuscular disease and gene expression.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kathryn Swoboda, MD, faculty emerita at Massachusetts General Hospital and a neurologist and rare disease specialist who's been working on SMA for nearly 30 years.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dutch neurologist Ewout Groen, PhD, a member of the Scientific Advisory Board of SMA Europe.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jaime Moore, MD, about weight loss drugs—specifically the role of GLP-1 receptor agonists in treating children with neuromuscular diseases and obesity.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric psychologist Natalie Truba, PhD, on the psychological aspects of gene therapy in neuromuscular disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Donna Shipp, a Boston-area nurse who was misdiagnosed with cancer, underwent surgery and later realized she had IgG4-RD. She now advocates on behalf of others with this rare disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Abby Bronson, vice president of patient advocacy at Edgewise Therapeutics, about a new effort to raise awareness of Becker muscular dystrophy as a disease distinct from Duchenne muscular dystrophy.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Allison Moore, founder and CEO of the Hereditary Neuropathy Foundation. Moore was the winner of the Muscular Dystrophy Association's 2026 Donavon Decker Legacy Award for Community Impact in Research.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Crowley, president and CEO of the Biotechnology Innovation Organization (BIO), founder of Amicus Therapeutics, and father of 2 children with Pompe disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Hyun Kim, MD, professor of medicine and director of the University of Minnesota's Interstitial Lung Disease Program, about idiopathic pulmonary fibrosis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Andrea Wilson-Woods, founder of Blue Faery: The Adrienne Wilson Liver Cancer Association—a nonprofit that advocates for patients with hepatocellular carcinoma.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michael Shilsky, MD, of the Yale School of Medicine, on potential therapies for Wilson disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Yen Chen, PhD, of the University of Michigan, on the subject of cognitive dysfunction or "brain fog" among people with scleroderma.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Danish hepatologist Aleksander Krag, MD, PhD, on the diagnosis and treatment of alpha-1 antitrypsin deficiency (AATD).

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Vesna Aleksovska, chair of the nonprofit organization Life With Challenges. Aleksovska, who has Gaucher disease, advocates for all rare disease patients in North Macedonia, a former Yugoslav republic.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Wesley Michael, founder of Rare Patient Voice. Since 2013, RPV has offered patients and caregivers opportunities to share their input with companies developing products to improve lives.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Katharine Provencher, director of patient advocacy at IgG4Ward!, a nonprofit that advocates for patients and caregivers affected by IgG4-RD.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Cecilia Dueñas, PsyD, on the PBC Research Foundation, and the discrimination and stigma she faced for years before getting diagnosed with the disease.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Golie-Lorenzo Green, whose lifelong battle with sickle cell disease led him to advocate for others with the debilitating illness.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jana Monaco, a NORD patient ambassador from Virginia who's made nationwide newborn screening her life's mission.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Alabama computer scientist Matthew Might, PhD, on how his son's death from a rare genetic disease led him to become an expert in AI and precision medicine.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sarah Chang, PhD, about the patient and caregiver burden associated with the ultra-rare mitochondrial disease TK2d.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews rheumatologist Jinoos Yazdany, MD, on the potentially catastrophic medical, ethical and legal consequences of relying too much on AI.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Barry Byrne, MD, PhD, an expert on Pompe disease and director of the University of Florida's Powell Gene Therapy Center.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Eric Matteson, MD, of the Mayo Clinic and winner of the American College of Rheumatology's 2025 Presidential Gold Medal.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Karen Smoot, a resident of New Mexico who has both idiopathic pulmonary fibrosis and pulmonary arterial hypertension. Karen is a patient ambassador for the Pulmonary Fibrosis Foundation.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Gabriela Romanow, founder of Boston, Massachusetts-based Rare Vision. The charity showcases works by artists with rare neuroimmune diseases such as NMOSD, MOGAD and transverse myelitis.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lisa Shea, director of global patient advocacy and engagement for immunology at Johnson & Johnson, about the experiences of patients affected by HDFN and FNAIT.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Nicole Lamanna, MD, a leukemia physician specializing in CLL treatment at Columbia University Medical Center in New York City.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews May Lee Tjoa, PhD, senior global medical affairs leader for Johnson & Johnson’s nipocalimab and maternal-fetal immunology division.

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Mascarenhas, MD, a professor with the Icahn School of Medicine at Mount Sinai in New York, on advancing care in myelofibrosis.