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LAL-D Features

Hagit Baris-Feldman
AATD Features, ALGS Features, CAD Features, CC Features, DMD Features, Features, GIST Features, HEM Features, IPF Features, LAL-D Features, LCFAOD Features, PAH Features, Pompe Features, SMA Features

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…

LAL-D therapy 4 brothers
Features, Human Interest, LAL-D Features

LAL-D Therapy Proves to Be a Lifesaver for 4 Michigan Brothers With the Rare Disease

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Lysosomal acid lipase deficiency (LAL-D) affects fewer than 1 in 50,000 people. But when it comes to rare diseases, Michigan schoolteacher Rebecca Trendy seems to have hit the jackpot.  All 4 of her sons have LAL-D — an inherited liver disease that renders the body unable to digest fats. “It’s been quite the medical journey…

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AATD Features, ALGS Features, CAD Features, CC Features, DMD Features, Features, GIST Features, HEM Features, Human Interest, IPF Features, LAL-D Features, LCFAOD Features, MTC Features, PAH Features

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…

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AATD Features, ALGS Features, CAD Features, CC Features, DMD Features, Features, GIST Features, HEM Features, Human Interest, IPF Features, LAL-D Features, LCFAOD Features, MS Features, MTC Features, PAH Features

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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