Rare Disease Advisor's Rare Care Podcast features exclusive interviews with experts and stakeholders from the rare disease community.
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Welcome to RARING, the voice of the rare disease community and a no-BS forum for patients and their families, rare disease patient organizations, and medical professionals. If you live with a rare disease or love someone who does, RARING is the podcast for you, by you, and with you every day. Tune in to hear leading experts discussing next-generation diagnostics and treatments. Learn from passionate nonprofit leaders talking about their highs and lows fighting for their communities. And laug ...
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We are three students at the Georgia Institute of Technology: Megen Wittling, Ann Johnson, and Jessie Morris. After taking a course called Health, Genes, and Society by Dr. Greg Gibson, we wanted to address some of these issues discussed in the course - personalized medicine and the field of quantified self. As our world enters a future with increased technology and information, we found it important to delve into the implications behind it and have created podcasts on several topics as a re ...
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126: An Interview With Pamela Gavin, New CEO of the National Organization for Rare Disorders (NORD)
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Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Pamela Gavin, the newly appointed CEO of the National Organization for Rare Disorders (NORD).By Rare Care Podcast
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125: An Interview With Patricia Weltin, Founder and CEO of Beyond The Diagnosis
17:59
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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.By Rare Care Podcast
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125: An Interview With Sarah Glass, PhD, of the n-Lorem Foundation
16:36
16:36
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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.By Rare Care Podcast
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124: An Interview with Dimitrios Karussis, MD, PhD, on Stem Cell Therapy for Multiple Sclerosis
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123: An Interview With Mary Morlino, Patient Navigator at the Undiagnosed Diseases Network Foundation
11:15
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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.By Rare Care Podcast
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122: An Interview WIth Tracy Sharp, a Patient With Lambert-Eaton Myasthenic Syndrome
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121: An Interview With University of Pennsylvania Neurologist and CIDP Expert Chafic Karam, MD
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120: An Interview with Avner Reshef, MD, Top Israeli Expert in Hereditary Angioedema
17:11
17:11
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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.By Rare Care Podcast
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119: An Interview With Pediatric Neurologist Edward Smith, MD, on the FDA Approval of Vamorolone for Boys With Duchenne Muscular Dystrophy
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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.By Rare Care Podcast
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118: An Interview With Sharon Hesterlee, PhD, Chief Research Officer at the Muscular Dystrophy Association
7:58
7:58
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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.By Rare Care Podcast
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117: An Interview With Ira Walker, the Muscular Dystrophy Association's 2024 National Ambassador, About Living With Spinal Muscular Atrophy
12:02
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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.By Rare Care Podcast
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116: An Interview With Seema Lalani, MD, Director of Project GIVE, a Pediatric Rare Disease Screening Program in the Rio Grande Valley of Texas
11:34
11:34
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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.…
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115: An Interview With Matt Trudeau of ITF Therapeutics, on Givinostat as a Therapy for Duchenne Muscular Dystrophy
9:52
9:52
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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.By Rare Care Podcast
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114: An Interview With Thomas Holm Pedersen, PhD, Cofounder and CEO of Denmark's NMD Pharma
14:34
14:34
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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.…
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113: An Interview With Courtney Silverthorn, MD, Associate VP of Science Partnerships at the Foundation for the National Institutes of Health
12:19
12:19
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12:19
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.By Rare Care Podcast
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112: An Interview With Cristol Barrett O'Loughlin, CEO of Raregivers
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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.By Rare Care Podcast
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111: An Interview With Omar Kamlin, MD, Senior Medical Director at Orphalan
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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.By Rare Care Podcast
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110: An Interview With Melinda Bachini, Chief Patient Officer at the Cholangiocarcinoma Foundation
18:20
18:20
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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.By Rare Care Podcast
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109: An Interview With Kyle Bryant, Director of the Ambassador Program at the Friedreich's Ataxia Research Alliance
9:03
9:03
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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.By Rare Care Podcast
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109: An Interview With Paul Bolno, MD, CEO of Wave Life Sciences
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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.By Rare Care Podcast
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An Interview With Chris Peetz, CEO of Mirum Pharmaceuticals
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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.By Rare Care Podcast
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107: An Interview With Thierry VandenDriessche, PhD, of the European Society of Gene & Cell Therapy
10:09
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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.By Rare Care Podcast
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106: An Interview With Brian O'Mahony, CEO of the Irish Haemophilia Society
8:38
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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.By Rare Care Podcast
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105: An Interview With Selene Capodarca, Global Study Coordinator for HD-Enroll
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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.By Rare Care Podcast
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104: An Interview With Professor Claire Booth, Cofounder of the AGORA Initiative
12:20
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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.By Rare Care Podcast
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103: An Interview With Dr. Paul Bolno, CEO of Wave Life Sciences
10:28
10:28
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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).By Rare Care Podcast
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102: An Interview With Margareth Ozelo, Director of the International Hemophilia Training Centre (IHTC) at Brazil's University of Campinas (UNICAMP)
6:55
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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.By Rare Care Podcast
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101: An Interview With Rob Haselberg, an Huntington Patient Advocate Who Has Tested Positive for the Disease
6:55
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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.By Rare Care Podcast
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100: An Interview With Herwig Lange, MD, President of the German Huntington Association
16:57
16:57
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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.By Rare Care Podcast
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99: An Interview With Cathleen Lutz, PhD, Vice President of the Jackson Laboratory's Rare Disease Transactional Center
14:20
14:20
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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…
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98: An Interview With Heather Stefanski, MD, PhD, Vice President of Medical Services at the National Marrow Donor Program's Be The Match
12:59
12:59
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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 …
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97: An Interview With Astri Arnesen, President and CEO of the European Huntington Association
16:59
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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.By Rare Care Podcast
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96: An Interview With Derek de Winter, MD, Coordinating Investigator of the DIONYSUS Study
11:05
11:05
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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).By Rare Care Podcast
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95: An Interview With Juan Valle, MB ChB, Chief Medical Officer of the Cholangiocarcinoma Foundation
13:58
13:58
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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.By Rare Care Podcast
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94: An Interview With Prader-Willi Syndrome Patient Advocate Paige Rivard
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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.By Rare Care Podcast
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93: An Interview With Italian Cholangiocarcinoma Expert Lorenza Rimassa, MD
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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.By Rare Care Podcast
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92: An Interview With Jennifer Miller, MD, A Leading Researcher in Prader-Willi Syndrome
16:14
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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.By Rare Care Podcast
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91: An Interview With Jennifer Knox, MD, Canada's Leading Expert in Cholangiocarcinoma
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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 …
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90: An Interview With Daniel Fischer, President and CEO of Tevard Biosciences
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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 …
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89: An interview with Damon Race, CEO of GeneVentive Therapeutics
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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.By Rare Care Podcast
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88: An Interview With Pediatrician and Medical Cannabis Specialist Bonni Goldstein, MD
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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.…
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87: An Interview With Jason Sicklick, MD, FACS, an Expert on Gastrointestinal Stromal Tumor
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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.…
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86: An Interview With Kattayoun Kordy, MD, Senior Director of Rare Disease Clinical Development/Immunology at Janssen
10:17
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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).…
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85: An Interview with Denise Scots-Knight, PhD, CEO of Mereo BioPharma
11:31
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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.By Rare Care Podcast
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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.By Rare Care Podcast
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83: An Interview with Kim Smith-Whitley, MD
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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.By Rare Care Podcast
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82: An Interview with Jason Tardio, Chief Operating Officer of Ovid Therapeutics
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Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jason Tardio, chief operating officer at Ovid Therapeutics,By Rare Care Podcast
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81: An Interview With Jennifer MacDonald, a Woman From Mexico With AATD
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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.By Rare Care Podcast
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80: An Interview With Mindy Henderson, Editor-in-Chief of MDA's Quest Magazine
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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.By Rare Care Podcast
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79: An Interview With DMD Patient Advocate Christopher Curran
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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.By Rare Care Podcast
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