77: An Interview With Luisa Leal, Founder and CEO of The Akari Foundation
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.
13m May 11, 2023
13m
76: An Interview With Julie Parsons, MD, Co-Director of the Neuromuscular Clinic at Children's Hospital Colorado
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 when Sarepta's gene therapy wins final approva...
16m May 04, 2023
16m
75: An Interview With Disabled Patient Advocate Christopher Rosa, PhD, President and CEO of the Viscardi Group
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 their wheelchairs during flight.
11m Apr 28, 2023
11m
74: An Interview With Matt Granato, President and CEO of the Pulmonary Hypertension Association
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.
14m Apr 18, 2023
14m
73: An Interview with Ron Bartek, Founding President of the Freidriech's Ataxia Research Alliance (FARA)
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.
12m Apr 12, 2023
12m
72: An Interview With Miranda Bradnick on How COVID-19 Has Affected Rare Disease Patients
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.
7m Apr 12, 2023
7m
71: An Interview With Jeff Szer, BMedSc, a Hematology Professor at Australia's Royal Melbourne Hospital
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).
14m Apr 04, 2023
14m
70: An Interview With Cheryl Schwartz, Takeda's Senior Vice-President of US Rare Disease Business
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.
7m Apr 04, 2023
7m
69: An Interview With John Berk, MD, Director of the Boston University School of Medicine's Localized Amyloid Clinic
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.
13m Mar 20, 2023
13m
68: An Interview With Çem Akin, MD, of the University of Michigan
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.
14m Mar 10, 2023
14m
67: An Interview With Dr. Abby Sandler of the National Cancer Institute
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dr. Abby Sandler of the National Cancer Institute.
11m Feb 28, 2023
11m
66: An Interview With George Goshua, MD, a Sickle Cell Expert at Yale University's School of Medicine.
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.
10m Feb 27, 2023
10m
65: An Interview With Arushi Khurana, MBBS, of the Mayo Clinic
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.
17m Feb 27, 2023
17m
64: An Interview With Catherine Broome, MD, an Associate Professor of Medicine at Georgetown Medstar University Hospital, in Washington DC
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.
9m Feb 14, 2023
9m
63: An Interview With Paul W. Noble, MD, of Cedars-Sinai Medical Center
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.
18m Feb 07, 2023
18m
62: An Interview With Federico Stella, MD, on the Use of Non-Restrictive Diets After Stem Cell Transplantation
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.
6m Feb 01, 2023
6m
61: An Interview With Abigail Jenkins, President and CEO of Gamida Cell Ltd.
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.
11m Jan 19, 2023
11m
60: An Interview With Peter Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD)
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.
11m Jan 11, 2023
11m
59: An Interview With Sara Rothschild, Executive Director of the Life Raft Group
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).
13m Jan 09, 2023
13m
58: An Interview With PJ Brooks, PhD, on the Bespoke Gene Therapy Consortium
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.
10m Dec 29, 2022
10m
57: An Interview With Scott Santarella, President and CEO of the Alpha-1 Foundation
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).
13m Dec 09, 2022
13m
56: An Interview With Lourdes Rocha-Nussbaum, Director of the Cholangiocarcinoma Foundation's Veterans Project
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.
11m Dec 05, 2022
11m
55: An Interview With Darlene Shelton, Founder and President of Danny's Dose Alliance
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Darlene Shelton, founder and president of Danny's Dose Alliance, a Missouri-based nonprofit that seeks to ensure proper emergency medical care for all patients with hemophilia and other bleeding disorders.
14m Nov 18, 2022
14m
54: An Interview With Aiwu Ruth He, MD, PhD, Associate Professor of Medicine at Medstar Georgetown University Hospital in Washington, D.C.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Aiwu Ruth He, MD, PhD, an associate professor of medicine at Medstar Georgetown University Hospital in Washington, D.C., and one of the principal investigators of the TOPAZ-1 clinical trial.
9m Nov 16, 2022
9m
53: An Interview With Dr. Brenda Wong, an expert on DMD and director of the MDA Care Center at the University of Massachusetts Chan Medical School
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Brenda Wong, MD, an expert on Duchenne muscular dystrophy and director of the MDA Care Center at the University of Massachusetts Chan Medical School in Worcester.
15m Nov 08, 2022
15m
52: An Interview With Randi Clites, Rare Disease Policy Director at the Little Hercules Foundation
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Randi Clites, rare disease policy director at the Ohio-based Little Hercules Foundation. This nonprofit's mission is to advocate for patients with Duchenne muscular dystrophy (DMD) and fight to get DMD included on every state's newborn screening panel.
9m Nov 01, 2022
9m
50: An Interview With Sumaira Ahmed, Founder and Executive Director of The Sumaira Foundation
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sumaira Ahmed, founder and executive director of The Sumaira Foundation, a Boston-based nonprofit organization that advocates on behalf of patients with neuromyelitis optica spectrum disorder (NMOSD) and related diseases.
15m Oct 12, 2022
15m
49: An Interview With Martin Mense, PhD, senior vice president of the Cystic Fibrosis Foundation
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Martin Mense, PhD, senior vice president of drug discovery at the Cystic Fibrosis Foundation, and head of the CFF's Therapeutics Lab in Lexington, Massachusetts.
13m Sep 22, 2022
13m
48: An Interview With Deanna Tucker, Medical Science Liaison and Team Leader at Sarepta Therapeutics
13m Sep 21, 2022
13m
47: An Interview With NMOSD Expert Dr. Michael Levy of Harvard Medical School
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michael Levy, MD, associate professor of neurology at Harvard Medical School in Boston, Massachusetts, and a leading expert on neuromyelitis optica spectrum disorder (NMOSD).
14m Sep 09, 2022
14m
46: An Interview With Jennifer Wallace Valdes, PT, Founder of the Duchenne Therapy Network
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Wallace Valdes, PT, founder of the Duchenne Therapy Network and physical therapist with CureDuchenne, on the importance of physical therapy and exercise in boys and young men with Duchenne muscular dystrophy.
8m Aug 31, 2022
8m
45: An Interview With Janet Lynch Lambert, CEO of the Alliance for Regenerative Medicine
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Janet Lynch Lambert, CEO of the Alliance for Regenerative Medicine (ARM). This Washington, DC-based nonprofit aims to bring safe and effective cell and gene therapies to patients around the world.
18m Aug 25, 2022
18m
44: An interview with Annie Kennedy, head of policy, advocacy, and patient engagement at the EveryLife Foundation for Rare Diseases
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Annie Kennedy, head of policy, advocacy, and patient engagement at the EveryLife Foundation for Rare Diseases. This Washington, DC-based nonprofit focuses on eliminating the diagnostic odyssey for rare disease, promoting regulations that speed up therapeutic development, and improving access to approved rare-disease therapies.
15m Aug 21, 2022
15m
43: An Interview With CureDuchenne Chief Scientific Officer Dr. Michael Kelly
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Michael Kelly, PhD, chief scientific officer at CureDuchenne, on the latest developments in exon skipping, gene therapy and other treatments for Duchenne muscular dystrophy.
11m Aug 15, 2022
11m
42: An Interview With LCFAOD Patient Advocate Eileen Sullivan Baker
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Eileen Sullivan Baker, an Ohio attorney whose son, John, has long chain fatty acid oxidation disorder. She has become an outspoken advocate for LCFAOD, in the absence of any formal patient advocacy organization for those with the disease.
16m Aug 04, 2022
16m
41: An interview with cattle rancher Paul Heaton of the Calves2Cure DMD initiative
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Paul Heaton, a Montana cattle rancher whose son has Duchenne, on his Calves2Cure DMD fundraising initiative.
9m Jul 27, 2022
9m
40: An interview with Emil Kakkis, MD, PhD, founder and CEO of Ultragenyx
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Emil Kakkis, MD, PhD, founder and CEO of San Francisco-based Ultragenyx, on the increasingly complex challenges facing developers of drugs to treat rare diseases.
14m Jul 20, 2022
14m
39: An interview With Muscular Dystrophy Researcher Pradeep Mammen, MD
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Pradeep Mammen, MD, professor of internal medicine and director of the Neuromuscular Cardiomyopathy Clinic at UT Southwestern in Dallas, Texas. Dr. Mammen, a speaker at the CureDuchenne 2022 Futures conference, has done extensive research on the issue of female carriers of muscular dystrophy.
13m Jul 13, 2022
13m
38: An interview with Diana Castro, MD, associate professor of pediatrics, neurology and neurotherapeutics at University of Texas Southwestern in Dallas
12m Jul 05, 2022
12m
37: An interview with motivational speaker Justin Skeesuck
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews wheelchair-bound Justin Skeesuck, who didn't let his progressive genetic disease stop him from crossing Spain's 500-mile Camino de Santiago. The adventure, with his best friend Patrick Gray, led to the book and movie "I'll Push You." Justin and Patrick were keynote speakers at the recent CureDuchenne 2022 Futures conference...
14m Jun 30, 2022
14m
36: An Interview With Shahid Khan, MD, of Imperial College London, on ICD-10 Coding for Cholangiocarcinoma
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Shahid Khan, MD, of Imperial College London, on why an update to ICD-10 coding is urgently needed for correct diagnosis of cholangocarcinoma.
16m Jun 21, 2022
16m
35: An interview with Marissa Penrod, founder of the Indiana-based nonprofit group Team Joseph
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Marissa Penrod, founder of the Indiana-based nonprofit group Team Joseph, which provides financial assistance to families like her own that have been affected by Duchenne muscular dystrophy.
12m Jun 15, 2022
12m
34: Dr. Mary Beth Scholand Discusses the Partnership Between CHEST and the Three Lakes Foundation
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Mary Beth Scholand, MD, director of the Interstitial Lung Disease Program at the University of Utah in Salt Lake City. Dr. Scholand discusses a newly announced initiative, "Bridging Specialties: Timely Diagnosis for ILD Patients," between the American College of Chest Physicians (CHEST) and the Three Lakes Foundation.
7m Jun 01, 2022
7m
33: An Interview with Josh Disbrow, CEO of Aytu Bio
Larry Luxner, senior correspondent for Rare Disease Advisor, interiews Josh Disbrow, CEO of Aytu Bio, a Colorado company developing potentially lifesaving products in the areas of pediatric onset and rare disease, particularly severe upper respiratory conditions.
13m May 25, 2022
13m
32: Solid Biosciences Founder Ilan Ganot Discusses His Quest for a Cure for DMD
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ilan Ganot, founder and CEO of Solid Biosciences in Cambridge, Massachusetts. The Israeli-born Ganot started his company in order to find a cure for his son, Eytani, who has DMD.
13m May 18, 2022
13m
31: An Interview With DMD Expert and Gene Therapy Pioneer Dr. Jerry Mendell
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jerry Mendell, MD, professor of neurology and pediatrics at Nationwide Children’s Hospital in Columbus, Ohio. Dr. Mendell is among the most prominent experts in Duchenne muscular dystrophy, and a pioneer in gene therapy. He met his first Duchenne patient in 1969.
13m May 10, 2022
13m
30: Dr. Henry Kaminski on the Difficulties of Treating Myasthenia Gravis
11m May 03, 2022
11m
29: Patient Advocate Laura McLinn Discusses Her Son's Journey With DMD
Larry Luxner, senior correspondent for Rare Disease Advisor, talks with Laura McLinn, the Indiana mom of 12-year-old Jordan McLinn, who has Duchenne muscular dystrophy. Jordan, who became the unofficial poster boy of the "Right to Try" movement, has just completed 5 years in a clinical trial for a novel exon 53 skipping therapy.
10m Apr 26, 2022
10m
28: Dr. Anthony Feinstein on His New Book About the Neurobehavioral Consequences of MS
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Anthony Feinstein, PhD, a professor of psychiatry at the University of Toronto and a world-renowned neuropsychiatrist. We talk to the South African-born Dr. Feinstein about his new book, "Mind, Mood and Memory: The Neurobehavioral Consequences of Multiple Sclerosis."
13m Apr 22, 2022
13m
27: Jay Griffin Discusses His Struggles Getting Treatment for His Son With DMD
12m Apr 11, 2022
12m