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.
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 (LCFAOD). She has become an outspoken advocate for people with LCFAOD, in the absence of any formal patient advocacy organization. Read the full story here: Son’s Illness Drives Ohio Attorney…
Which of the statements below about the signs and symptoms of Alagille syndrome (ALGS) is incorrect? 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 the symptoms of Alagille syndrome.
Which of the statements below about the diagnosis of cold agglutinin disease (CAD) 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 diagnosis of CAD.
Which of the statements below about the diagnosis of hereditary angioedema (HAE) 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 diagnosis of hereditary angioedema.
Which of the statements below about vaso-occlusive crises in sickle cell disease (SCD) is incorrect? 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 comorbidities of sickle cell disease.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Deanna Tucker, PharmD, a medical science liaison team leader at Sarepta Therapeutics, on the company’s efforts to develop a gene therapy for Duchenne muscular dystrophy. Read the full story here: Sarepta Simultaneously Pursues Exon Skipping, PPMO, and Gene Therapies for DMD
Is the statement below about the pathophysiology of Pompe disease 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 Pompe disease pathophysiology.
Which of the statements below about LAL-D diagnosis 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 LAL-D here.
Which of the statements below about the types of long chain fatty acid oxidation disorder (LCFAOD) 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 types of LCFAOD.
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