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AATD Features

Hagit Baris-Feldman
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Israel Emerges as Global Powerhouse in Rare Disease Research

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TEL AVIV, Israel — On the 6th floor of Schneider Children’s Hospital in the Tel Aviv suburb of Petah Tikva, 30 children with Alagille syndrome and 2 with lysosomal acid lipase deficiency (LAL-D) receive specialized treatment from a trio of pediatric hepatologists. Across town, at Sheba Medical Center’s Edmond and Lily Safra Children’s Hospital, pediatric…

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Researchers Exploring Multiple Mechanisms of Action for Treating AATD

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Faron Schonfeld, a successful investment portfolio manager from Philadelphia, Pennsylvania, had for years suffered chronic coughing and sinus infections that only seemed to get worse. He was finally referred to a pulmonologist at National Jewish Health in Denver, Colorado.  That’s where he got the diagnosis: Schonfeld had alpha-1 antitrypsin deficiency (AATD, or simply alpha-1) —…

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Emerging Therapies, COVID-19 Highlight Alpha-1 Foundation’s Upcoming ‘Virtual Education Day’

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Alpha-1 antitrypsin deficiency (AATD) is among the most common life-threatening genetic diseases to affect adult Caucasians worldwide. Yet for years, there’s only been one way to treat it: augmentation therapy. All 4 augmentation therapy products approved by the US Food and Drug Administration (FDA) involve taking alpha-1 antitrypsin protein (AAT) from the plasma of healthy…

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NORD Recognizes Progress in Fight Against Rare Disease With 2021 Rare Impact Awards

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Under COVID-19’s lingering shadow, the National Organization of Rare Disorders (NORD) has presented its 2021 Rare Impact Awards to 24 pharmaceutical firms, researchers, politicians, and nonprofit groups for their efforts over the past year on behalf of patients with such illnesses. The virtual ceremony was hosted by John Whyte, MD, MPH, chief medical officer at WebMD, with commentary…

Alpha-1 Antitrypsin Deficiency & COVID-19
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Examining the Links Between SARS-CoV-2 and Alpha-1 Antitrypsin Deficiency

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Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection can cause a variety of symptoms, and people with chronic lung disease including chronic obstructive pulmonary disease (COPD), chronic liver disease especially cirrhosis, diabetes, and cardiovascular disease are among those who are more prone to develop severe infection. We now know that severe SARS-CoV-2 infection is mainly…

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FDA, CDC Urge COVID-19 Vaccines for Rare Disease Patients on NORD Webinar

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As the super-contagious Delta variant of COVID-19 sweeps across the globe, driving up infection rates as well as deaths, top US health officials are urging everyone 12 and older — including the country’s 30 million or so rare disease patients — to get vaccinated quickly. Some 185 million people have been infected, and nearly 4…

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