Dr. Naftali Kaminski Discusses New Consortium for Pulmonary Fibrosis
Larry Luxner, senior correspondent for Rare Disease Advisor, talks with pulmonologist and Yale University School of Medicine professor Naftali Kaminski, MD.
Podcasts
Dr. Durhane Wong-Rieger on the Challenges Faced by Rare Disease Patients in Canada
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Durhane Wong-Rieger, PhD, president and CEO of the Canadian Organization for Rare Disorders (CORD) and chair of Rare Diseases International (RDI). Dr. Wong-Rieger talks about the challenges of diagnosing and treating diseases in Canada, as well as advancing access to therapies around the globe. Read the…
An Update on LAL-D With Dr. Paul Martin of Nationwide Children’s Hospital
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Paul T. Martin, PhD, principal investigator at the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio. Dr. Martin has been at the forefront of research into potential gene therapies for lysosomal acid lipase deficiency, or LAL-D. Read the full article: Gene Therapy May…
A Discussion on Medullary Thyroid Carcinoma With Dr. David Goldenberg
In this episode of the Rare Care podcast, Larry Luxner interviews surgical oncologist David Goldenberg, MD, FACS, about medullary thyroid carcinoma.
Test Your Knowledge About the Symptoms of Patients With AATD
Which of the statements below about symptoms in alpha-1 antitrypsin deficiency (AATD) are correct? Select one of the options, then submit your answers to see your score and the average score for everyone who has taken the quiz. Read more about AATD symptoms here.
Test Your Knowledge About the Differential Diagnosis of Patients With LCFAOD
Which of the statements below about the diagnosis of patients in long chain fatty acid oxidation disorder (LCFAOD) are accurate? Answer “true” or “false,” then submit your answers to see your score and the average score for everyone who has taken the quiz. Read more about LCFAOD differential diagnosis here.
The Pursuit of Redosable Gene Therapies for Hemophilia With Dr. Doug Kerr
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Doug Kerr, MD, PhD, chief medical officer at Generation Bio in Cambridge, Massachusetts. The company is aggressively pursuing redosable gene therapies for hemophilia and other rare diseases. Read the full article: Generation Bio Pursues Redosable Gene Therapy for Hemophilia A
Test Your Knowledge of the Complications of LAL-D
Take our quick quiz on statements about complications in lysosomal acid lipase deficiency.
Perspectives on Long Chain Fatty Acid Oxidation Disorder With Dr. Jerry Vockley
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jerry Vockley, MD, PhD, chief of medical genetics at Pennsylvania’s UPMC Children’s Hospital of Pittsburgh. Dr. Vockley is also the founder of INFORM, the International Network for Fatty Acid Oxidation Research and Management, which will hold its next annual conference virtually October 27-28, 2021. Read the…
Test Your Knowledge About the Prognosis of Patients With AATD
Which of the statements below about prognosis in alpha-1 antitrypsin deficiency (AATD) are accurate? Answer “true” or “false,” then submit your answers to see your score and the average score for everyone who has taken the quiz. Read more about AATD prognosis here.
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