Podcast Rare Care

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).Oleh Rare Care Podcast

In this episode of the I Care for Rare podcast hosts Sandra Markus and Sherrilynne Starkie interview Tammie Moretton, a parent who has journeyed the intricate road of caring for and raising a child with complex needs, and who sheds light on the challenges faced by such families in Canada. I Care for Rare is more than just a podcast; it's a social a…

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.Oleh 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.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Herwig Lange, MD, a neurologist who has been researching Huntington disease since 1969. Dr. Lange, a proponent of non-drug therapy to improve the lives of HD patients, is also president of the German Huntington Association.Oleh Rare Care Podcast

Step into the world of rare diseases and learn about the challenges faced by families across Canada. In this inaugural episode of the I Care for Rare podcast host Sherrilynne Starkie welcomes Sandra Markus, the visionary behind the I Care for Rare campaign, to share her personal journey and the mission to create a collective voice for individuals, …

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Cathleen Lutz, PhD, vice president of the Jackson Laboratory's Rare Disease Transactional Center in Bar Harbor, Maine. Dr. Lutz is leading efforts to implement the latest genomic editing techniques to address the actual genetic defect in Friedreich ataxia, with the goal of tran…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Heather Stefanski, MD, PhD, vice president of medical services at the National Marrow Donor Program / Be The Match. Previously, she was an associate professor of pediatrics at the University of Minnesota. Dr. Stefanski's research focuses on children with life-threatening blood …

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Astri Arnesen, president and CEO of the European Huntington Association (EHA), about current research trends and the potential for gene therapy to treat Huntington disease ahead of EHA's 2023 conference in Blankenberge, Belgium.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Derek de Winter, coordinating investigator of the DIONYSUS study, a Dutch-based international retrospective registry on hemolytic disease of the fetus and newborn (HDFN).Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Juan Valle, MB ChB, about his appointment as the Cholangiocarcinoma Foundation's first-ever chief medical officer and what it means for patients with the disease.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Paige Rivard, past president of the Prader-Willi Syndrome Association USA, on the extreme challenges faced by parents of children with this disease.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lorenza Rimassa, MD, associate professor of medical oncology at Italy’s Humanitas University and Humanitas Research Hospital, about current research on treatment options for cholangiocarcinoma.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Miller, MD, a professor of pediatric endocrinology at the University of Florida who specializes in treating children with Prader-Willi syndrome and other causes of excessive weight gain in childhood.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Knox, MD, at the 25th World Congress on Gastrointestinal Cancer in Barcelona, Spain. Dr. Knox, considered Canada's top expert in cholangiocarcinoma, ia a professor of medicine at the University of Toronto, and a staff medical oncologist at the Princess Margaret Cancer …

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Daniel Fischer, president and CEO of Tevard Biosciences, which is "Dravet" spelled backwards. Fischer and his business partner, Warren Lammert—both fathers of girls with Dravet syndrome—are developing technologies to modulate RNA function to treat rare and severe diseases that …

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Damon Race, CEO of GeneVentive Therapeutics. The North Carolina-based startup hopes to develop a universal gene therapy that will benefit hemophilia A and B patients with inhibitors.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Bonni Goldstein, MD, a Los Angeles-based pediatrician and medical cannabis specialist who has treated more than 18,000 patients, 80% of whom are children with various forms of epilepsy, including Dravet and Lennox-Gastaut syndromes.…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jason Sicklick, MD, FACS, an expert on gastrointestinal stromal tumor (GIST), ahead of July 13, GIST Awareness Day. Dr. Sicklick's laboratory focuses on the molecular mechanisms of GIST development and drug resistance in advanced GIST.…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kattayoun Kordy, MD, senior director of rare disease clinical development/immunology at Janssen. She discusses her company's clinical trials for nipocalimab, an intravenous infusion that aims to treat hemolytic disease of the fetus and newborn (HDFN).…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Denise Scots-Knight, PhD. Her UK-based company, Mereo BioPharma, is developing therapies for alpha-1 antitrypsin deficiency.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews psychologist Al Freedman, PhD, whose late son Jack had spinal muscular atrophy. Dr. Freedman specializes in counseling families of those affected by rare disease.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kim Smith-Whitley, MD, a pediatric hematologist and top Pfizer execuive specializing in the treatment of sickle cell disease.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jason Tardio, chief operating officer at Ovid Therapeutics,Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer MacDonald—who has alpha-1 antitrypsin deficiency— about the difficulties of being a rare disease patient in Mexico.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Mindy Henderson, an SMA patient who is editor-in-chief of Quest, the quarterly magazine of the Muscular Dystrophy Association.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Christopher Curran, who along with his wife, Jessica, founded Kindness Over Muscular Dystrophy to advocate for patients like their 12-year-old son, Conner, who has Duchenne muscular dystrophy.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Leah Zelaya, a 2023 national ambassador of the Muscular Dystrophy Association. The 15-year-old aspiring actress from Brooklyn, New York, has an ultra-rare form of spinal muscular atrophy (SMA) known as scapulopereneal SMA.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Luisa Leal, founder and CEO of The Akari Foundation, a Texas-based nonprofit that advocates on behalf of Spanish-speaking families affected by Duchenne muscular dystrophy.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric neurologist Julie Parsons, MD, of Children's Hospital Colorado in Denver. An expert on both muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD), Dr. Parsons discusses how the nation's healthcare system must prepare for an avalanche of Duchenne patients if and…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Christopher Rosa, PhD, president and CEO of the Viscardi Group. Dr. Rosa, who has limb-girdle muscular dystrophy, is a longtime advocate for the disabled community. His chief mission now is pushing legislation that will require U.S. airlines to let disabled passengers remain in…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Matt Granato, president and CEO of the Pulmonary Hypertension Association, on World PH Day 2023—which takes place May 5—and the urgency of creating awareness about PAH, a frequently misdiagnosed and potentially fatal disease.Oleh Rare Care Podcast

Larry Luxner, senior corespondent for Rare Disease Advisor, interviews Ron Bartek, founding president of the Freidriech's Ataxia Research Alliance (FARA), about the hope generated by the recent FDA approval of Reata's omaveloxolone (Skyclarys)—the first-ever therapy to treat this rare neuromuscular disease.…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Miranda Bradnick, the mother of 3 children with Alagille syndrome. Among her biggest concerns: how COVID-19 has changed the lives of her family and others affected by rare disease, 3 years after the World Health Organization declared a pandemic.…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jeff Szer, BMedSc, a hematology professor at Australia's Royal Melbourne Hospital and secretary of the UK-based International PNH Interest Group, which is organizing its first-ever scientific symposium on paroxysmal nocturnal hemogloblnuria (PNH).…

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Cheryl Schwartz, Takeda's senior vice-president of US rare disease business, on the recent approval of lanadelumab-flyo (Takhzyro®) for hereditary angioedema.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Berk, MD, director of the Boston University School of Medicine's Localized Amyloid Clinic, on the latest therapeutic options for hereditary ATTR amyloidosis.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Çem Akin, MD, a specialist in allergy and immunology at the University of Michigan, about various treatment options for systemic mastocytosis.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dr. Abby Sandler of the National Cancer Institute.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Yale University hematologist George Goshua, MD, who has completed a cost analysis of gene therapy versus standard of care in patients with sickle cell disease.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Arushi Khurana, MBBS, a hematologist and lead author of a study showing that minorities are less likely to receive front-line therapy for diffuse large B-cell lymphoma than white patients.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Catherine Broome, MD, on the relative benefits to quality of life by treating immune thrombocytopenia patients with efgartigimod.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Paul W. Noble, MD, director of the Women’s Guild Lung Insitute at Cedars-Sinai Medical Center in Los Angeles, about his work exploring zinc as a potential therapy for idiopathic pulmonary fibrosis.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Federico Stella, MD, on the controversial practice of severely restricting the diets of patients who have just undergone stem cell transplants.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Abigail Jenkins, president and CEO of Gamida Cell Ltd., on the company's development of omidubicel as an alternative to umbilical cord blood in stem cell transplants for myelofibrosis and other patients.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Peter Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD), on the 40th anniversary of the landmark Orphan Drug Act.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sara Rothschild, the new executive director of the Life Raft Group, the nation's chief nonprofit advocating on behalf of patients with gastrointestinal stromal tumor (GIST).Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Philip J. Brooks, PhD, acting director of the Division of Rare Diseases Research Innovation at NCATS. Dr. Brooks describes the mission of the Bespoke Gene Therapy Consortium, which recently marked its first anniversary.Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, talks with Scott Santarella, the new president and CEO of the Miami-based Alpha-1 Foundation—the nation's largest organization working on behalf of patients with alpha-1 antitrypsin deficiency (AATD).Oleh Rare Care Podcast

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Lourdes Rocha-Nussbaum, director of the Cholangiocarcinoma Foundation's Veterans Project. This program explores the relatively high incidence of bile duct cancer among Vietnam War veterans and care options for such patients.Oleh Rare Care Podcast