125: An Interview With Patricia Weltin, Founder and CEO of Beyond The Diagnosis
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.
18m Jun 03, 2024
18m
125: An Interview With Sarah Glass, PhD, of the n-Lorem Foundation
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.
17m May 30, 2024
17m
124: An Interview with Dimitrios Karussis, MD, PhD, on Stem Cell Therapy for Multiple Sclerosis
15m May 28, 2024
15m
123: An Interview With Mary Morlino, Patient Navigator at the Undiagnosed Diseases Network Foundation
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.
11m May 16, 2024
11m
122: An Interview WIth Tracy Sharp, a Patient With Lambert-Eaton Myasthenic Syndrome
14m May 07, 2024
14m
121: An Interview With University of Pennsylvania Neurologist and CIDP Expert Chafic Karam, MD
4m May 03, 2024
4m
120: An Interview with Avner Reshef, MD, Top Israeli Expert in Hereditary Angioedema
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.
17m Apr 25, 2024
17m
119: An Interview With Pediatric Neurologist Edward Smith, MD, on the FDA Approval of Vamorolone for Boys With Duchenne Muscular Dystrophy
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.
16m Apr 25, 2024
16m
118: An Interview With Sharon Hesterlee, PhD, Chief Research Officer at the Muscular Dystrophy Association
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.
8m Apr 15, 2024
8m
117: An Interview With Ira Walker, the Muscular Dystrophy Association's 2024 National Ambassador, About Living With Spinal Muscular Atrophy
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.
12m Apr 05, 2024
12m
116: An Interview With Seema Lalani, MD, Director of Project GIVE, a Pediatric Rare Disease Screening Program in the Rio Grande Valley of Texas
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.
12m Apr 02, 2024
12m
115: An Interview With Matt Trudeau of ITF Therapeutics, on Givinostat as a Therapy for Duchenne Muscular Dystrophy
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.
10m Mar 22, 2024
10m
114: An Interview With Thomas Holm Pedersen, PhD, Cofounder and CEO of Denmark's NMD Pharma
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.
15m Mar 14, 2024
15m
113: An Interview With Courtney Silverthorn, MD, Associate VP of Science Partnerships at the Foundation for the National Institutes of Health
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.
12m Mar 06, 2024
12m
112: An Interview With Cristol Barrett O'Loughlin, CEO of Raregivers
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.
13m Feb 28, 2024
13m
111: An Interview With Omar Kamlin, MD, Senior Medical Director at Orphalan
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.
15m Feb 23, 2024
15m
110: An Interview With Melinda Bachini, Chief Patient Officer at the Cholangiocarcinoma Foundation
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.
18m Feb 02, 2024
18m
109: An Interview With Kyle Bryant, Director of the Ambassador Program at the Friedreich's Ataxia Research Alliance
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.
9m Jan 22, 2024
9m
109: An Interview With Paul Bolno, MD, CEO of Wave Life Sciences
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.
13m Jan 04, 2024
13m
An Interview With Chris Peetz, CEO of Mirum Pharmaceuticals
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.
14m Dec 27, 2023
14m
107: An Interview With Thierry VandenDriessche, PhD, of the European Society of Gene & Cell Therapy
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.
10m Dec 14, 2023
10m
106: An Interview With Brian O'Mahony, CEO of the Irish Haemophilia Society
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.
9m Dec 14, 2023
9m
105: An Interview With Selene Capodarca, Global Study Coordinator for HD-Enroll
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.
11m Dec 07, 2023
11m
104: An Interview With Professor Claire Booth, Cofounder of the AGORA Initiative
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.
12m Dec 05, 2023
12m
103: An Interview With Dr. Paul Bolno, CEO of Wave Life Sciences
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).
10m Nov 21, 2023
10m
102: An Interview With Margareth Ozelo, Director of the International Hemophilia Training Centre (IHTC) at Brazil's University of Campinas (UNICAMP)
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.
7m Nov 15, 2023
7m
101: An Interview With Rob Haselberg, an Huntington Patient Advocate Who Has Tested Positive for the Disease
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.
7m Nov 14, 2023
7m
100: An Interview With Herwig Lange, MD, President of the German Huntington Association
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.
17m Nov 03, 2023
17m
99: An Interview With Cathleen Lutz, PhD, Vice President of the Jackson Laboratory's Rare Disease Transactional Center
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 translating it to the clinic.
14m Oct 05, 2023
14m
98: An Interview With Heather Stefanski, MD, PhD, Vice President of Medical Services at the National Marrow Donor Program's Be The Match
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 and immune system disorders.
13m Sep 21, 2023
13m
97: An Interview With Astri Arnesen, President and CEO of the European Huntington Association
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.
17m Sep 15, 2023
17m
96: An Interview With Derek de Winter, MD, Coordinating Investigator of the DIONYSUS Study
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).
11m Sep 13, 2023
11m
95: An Interview With Juan Valle, MB ChB, Chief Medical Officer of the Cholangiocarcinoma Foundation
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.
14m Sep 01, 2023
14m
94: An Interview With Prader-Willi Syndrome Patient Advocate Paige Rivard
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.
17m Aug 29, 2023
17m
93: An Interview With Italian Cholangiocarcinoma Expert Lorenza Rimassa, MD
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.
16m Aug 21, 2023
16m
92: An Interview With Jennifer Miller, MD, A Leading Researcher in Prader-Willi Syndrome
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.
16m Aug 11, 2023
16m
91: An Interview With Jennifer Knox, MD, Canada's Leading Expert in Cholangiocarcinoma
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 Center.
17m Aug 03, 2023
17m
90: An Interview With Daniel Fischer, President and CEO of Tevard Biosciences
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 are usually not amenable to traditional gene th...
16m Jul 26, 2023
16m
89: An interview with Damon Race, CEO of GeneVentive Therapeutics
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.
13m Jul 19, 2023
13m
88: An Interview With Pediatrician and Medical Cannabis Specialist Bonni Goldstein, MD
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.
12m Jul 14, 2023
12m
87: An Interview With Jason Sicklick, MD, FACS, an Expert on Gastrointestinal Stromal Tumor
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.
14m Jul 07, 2023
14m
86: An Interview With Kattayoun Kordy, MD, Senior Director of Rare Disease Clinical Development/Immunology at Janssen
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).
10m Jun 30, 2023
10m
85: An Interview with Denise Scots-Knight, PhD, CEO of Mereo BioPharma
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.
12m Jun 21, 2023
12m
84: Rare Disease Advisor
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.
15m Jun 16, 2023
15m
83: An Interview with Kim Smith-Whitley, MD
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.
15m Jun 09, 2023
15m
82: An Interview with Jason Tardio, Chief Operating Officer of Ovid Therapeutics
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jason Tardio, chief operating officer at Ovid Therapeutics,
13m Jun 06, 2023
13m
81: An Interview With Jennifer MacDonald, a Woman From Mexico With AATD
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.
13m May 30, 2023
13m
80: An Interview With Mindy Henderson, Editor-in-Chief of MDA's Quest Magazine
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.
14m May 30, 2023
14m
79: An Interview With DMD Patient Advocate Christopher Curran
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.
15m May 19, 2023
15m
78: An Interview With Leah Zelaya, a Muscular Dystrophy Association 2023 national ambassador
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.
6m May 17, 2023
6m