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…
Take our quick quiz on statements about complications in lysosomal acid lipase deficiency.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Hagit Baris-Feldman, MD, director of the Genetics Institute at Israel’s Tel Aviv Sourasky Medical Center. Dr. Baris-Feldman, who also chairs the Israel Medical Genetics Association and is an associate professor at Tel Aviv University’s Sackler Faculty of Medicine, talks about why Israel has emerged as a…
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Edward Neilan, MD, chief medical officer at the National Organization for Rare Disorders (NORD). This is a new position at NORD, which serves as the umbrella organization for some 330 disease-specific patient advocacy groups. NORD also sponsors the annual Rare Diseases and Orphan Products Breakthrough Summit,…
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