Which of the following investigations is the least necessary in an alpha-1 antitrypsin deficiency (AATD) diagnostic workup? Select one of the options for the question below, then submit your answer to see your score and the average score for everyone who has taken the quiz. Read more about AATD diagnosis.
Which of the statements below about the epidemiology of alpha-1 antitrypsin deficiency (AATD) are correct? Select one of the options for each question, then submit your answers to see your score and the average score for everyone who has taken the quiz. Read more about the epidemiology of AATD.
Is the statement below about the diagnosis of alpha-1 antitrypsin deficiency (AATD) correct? Answer true or false. Select a response to the question, then submit your answer to see your score and the average score for everyone who has taken the quiz. Read more about AATD diagnosis here. Reference Sandhaus R, Gerard T. The diagnosis…
Rare Disease Advisor senior correspondent Larry Luxner interviews Ultragenyx CEO Dr. Emil Kakkis on the challenges of developing rare disease drugs.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Josh Disbrow, CEO of Colorado-based Aytu BioPharma.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Amir London, CEO of Kamada, a major biopharmaceutical company that has a therapy for alpha-1 antitrypsin deficiency (AATD) approved by the US Food and Drug Administration and is now pursuing an inhaled alpha-1 antitrypsin (AAT) drug for the same disease. Read the full story: Inhaled Therapy…
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.
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.
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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