Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jean Elwing, MD, a professor of medicine and director of the Pulmonary Hypertension Program at Ohio's University of Cincinnati.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare DIsease Advisor, interviews Payel Gupta, MD, on social media platforms such as TikTok, Facebook and YouTube, and their importance in reaching today's public—and fighting online misinformation.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sandy Siegel, founder and president of the Siegel Rare Neuroimmune Association. Siegel started the charity 30 years ago with his wife, Pauline, after she was diagnosed with transverse myelitis. After Pauline's death in 2017, the organization expanded to include five other rare …

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Anastasia Vishnevetsky, MD, MPH, of Massachusetts General Hospital and Harvard Medical School. Dr. Vishnevetsky is heading a groundbreaking clinical trial of the cannabinoid spray nabiximols in treating the symptoms of NMOSD.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ignacio Muñoz-Sanjuan, PhD, the president and founder of Factor-H—a Los Angeles-based nonprofit organization that works to alleviate extreme poverty among people with Huntington disease in Venezuela, Colombia, and Peru.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Amy Case, MD, chief medical officer of the Pulmonary Fibrosis Foundation, about the importance of passing the bipartisan Supplemental Oxygen Access Reform (SOAR) Act, which is currently stalled in Congress.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Steve Van Wormer, cofounder and director of the Phaware Global Association. Van Wormer became a patient advocate after his late son, Lucas, was diagnosed with pulmonary hypertension at age 4.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Victor Test, MD, an expert in pulmonary arterial hypertension (PAH). Dr. Test is a professor of medicine and chair of the pulmonary vascular disease program at Texas Tech School of Medicine in Lubbock.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pulmonologist Drew Harris, MD, an associate professor of medicine at the University of Virginia in Charlottesville. Dr. Harris is also medical director of the Black Lung Program at Stone Mountain Health Services, in the heart of southwestern Virginia's coal-mining industry.…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Mahesh Desai, PhD, of the Luxembourg Institute of Health’s Department of Infection and Immunity. Dr. Desai discusses how healthy gut bacteria may reduce the side effects of chemotherapy in cancer patients.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Karin Hoelzer, DVM, PhD, senior director of policy and regulatory affairs at the National Organization for Rare Disorders (NORD). Dr. Hoelzer discusses the importance of convincing the US Congress to renew the Rare Pediatric Disease Priority Review Voucher Program, which is set…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Susan Ward, PhD, founder and executive director of the Collaborative Trajectory Analysis Project (cTAP), which seeks to broaden patient eligibility for clinical trials in Duchenne muscular dystrophy.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Taylor Kane, founder and executive director of 'Remember the Girls.' The charity advocates for female carriers of X-linked rare disorders including Duchenne muscular dystrophy and hemophilia.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews neurologist Jeffrey A. Cohen, MD, of Ohio's Cleveland Clinic on the latest developments in cellular therapies to treat multiple sclerosis.Door Rare Care Podcast

Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jacquelyn Bainbridge, DPharm, a neurology professor at the University of Colorado in Aurora, on the benefits of medical cannabis in treating multiple sclerosis.Door Rare Care Podcast

Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Flavia Nelson, MD, director of the Multiple Sclerosis Center of Excellence at the University of Miami in Florida, on the difficulties of diagnosing neuromyelitis optica spectrum disorder.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kevin M. Flanigan, MD, director of the Center for Gene Therapy at the Abigail Wexner Research Instute of Nationwide Children’s Hospital in Columbus, Ohio. The subject of our talk is exon skipping therapies for Duchenne muscular dystrophy.…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Durresamin Khan, the Pakistani mother of a boy with Duchenne muscular dystrophy, about the difficulties of obtaining treatment for DMD patients who are ineligible for gene therapy.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jenny Huang, whose son, Benjamin Lou, is an award-winning math major at MIT who also has spinal muscular atrophy.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Omer Abdul Hamid, MD, a neurologist at Nemours Children's Hospital in Orlando, Florida, on gene therapy's potential to treat Duchenne muscular dystrophy.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Pamela Gavin, the newly appointed CEO of the National Organization for Rare Disorders (NORD).Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Patricia Weltin,founder and CEO of Beyond the Diagnosis—a nonprofit that uses art to raise public awareness of children with ultra-rare diseases.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sarah Glass, PhD, chief operating officer of the n-Lorem Foundation—and the mother of a boy with an ultra-rare disease.Door Rare Care Podcast

Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Mary Morlino, who has sarcoidosis and is the patient navigator for the Undiagnosed Diseases Network Foundation.Door Rare Care Podcast

Door Rare Care Podcast

Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Israeli expert Avner Reshef, MD, director of the Allergy, Immunology, and Angioedema Center at Barzilai University Medical Center in Ashkelon, only a few miles from the border with war-torn Gaza.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric neurologist Edward Smith, MD, about the recent approval of vamorolone, a synthetic corticosteroid, in boys with Duchenne muscular dystrophy.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sharon Hesterlee, PhD, chief research officer at the Muscular Dystrophy Association, about the recent proliferation of gene therapies and other treatments for neuromuscular diseases.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ira Walker, the Muscular Dystrophy Association's 2024 National Ambassador, at the MDA's 2024 Clinical & Scientific Conference in Orlando, Florida. Walker, 39, has spinal muscular atrophy (SMA) type 2.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Seema Lalani, MD, of Baylor College of Medicine in Houston, Texas. Dr. Lalani directs Project GIVE (Genetic Inclusion by Virtual Evaluation), a pediatric screening program in the impoverished 4-county Rio Grande Valley region of southern Texas.…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Matt Trudeau, head of ITF Therapeutics—a division of Italy's Italfarmaco—on treating Duchenne muscular dystrophy with givinostat, a histone deacetylase inhibitor.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Thomas Holm Pedersen, PhD, cofounder and chief executive of NMD Pharma, a Danish company that's pursuing the use of chloride channel 1 (ClC-1) as a novel target for restoring muscle function in both myasthenia gravis and spinal muscular atrophy.…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Courtney Silverthorn, MD, of the Foundation for the National Institutes of Health (NIH) about the NIH's Bespoke Gene Therapy Consortium.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Cristol Barrett O'Loughlin, founder and CEO of Raregivers—a nonprofit organization that offers mental health and wellness services to those caring for people with rare diseases.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Omar Kamlin, MD, senior medical director at French drugmaker Orphalan, whose therapy, Cuvrior, treats Wilson disease.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Melinda Bachini, chief patient officer at the Cholangiocarcinoma Foundation, about a new study detailing the burden on caregivers of people with cholangiocarcinoma.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kyle Bryant, director of the Ambassador Program at the Friedreich's Ataxia Research Alliance (FARA), and founder and director of rideATAXIA.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Paul Bolno, MD, the CEO of Wave Life Sciences, whose investigational therapy WVE-003 is a potential treatment for Huntington disease.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Chris Peetz, CEO of Mirum Pharmaceuticals, about the growing number of treatments for pruritis (severe itch), one of the worst symptoms associated with Alagille syndrome.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Belgian molecular biologist Thierry VandenDriessche, PhD, president of the local organizing committee of the European Society of Gene & Cell Therapy.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Brian O'Mahony, president of the Irish Haemophilia Society and one of the few hemophilia B patients in Europe to receive gene therapy.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Selene Capodarca. This Italian expert in pharmaceutical chemistry and technology, is the global study director for Enroll-HD, with 30,000 patients the world's largest observational study in Huntington disease.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, talks with Professor Claire Booth of University College London and cofounder of the AGORA Initiative, which aims to tackle the growing economic hurdles that prevent lifesaving gene therapies from reaching children who need them the most.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Paul Bolno, MD, the CEO of Wave Life Sciences, whose investigational therapy WVE-006 is a potential treatment for the liver disease associated with alpha-1 antitrypsin disorder (AATD).Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Brazilian bleeding disorders expert Margareth Ozelo, who has been researching gene therapies for hemophilia for the past 25 years.Door Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Rob Haselberg, a Dutch patient advocate for Huntington disease who is heatlhy now, but will develop HD later in life after having tested positive for the incurable, progressive illness.Door Rare Care Podcast