Takeda and its collaborator, Arrowhead Pharmaceuticals, recently announced results from AROAAT-2002 of the investigational drug fazirsiran. The drug is an RNA interference therapeutic developed to lessen alpha-1 antitirypsin protein…
22-year-old Ruchita Sahukari is currently studying Literature and Sociology at the Indira Gandhi National Open University (IGNOU). Her journey to pursuing her degree began with the assistance of non-governmental…
Written by Becky Tilley My name is Becky Tilley and I am a rare woman, mama, writer and advocate. Also am proud wife to my husband Carl and mother to…
Shortly after he was born, Asa Burnside was diagnosed with Niemann-Pick disease type A, a rare genetic metabolic disorder. Since then, Asa has brought so much joy to his family’s…
In September 2020, Amelia Bradford turned eight months old. Her family loved their fun, wiggly, and happy baby. But then Amelia’s mother Danielle noticed something concerning: a facial bruise around…
Have you ever heard of Orphan Drug designation? In the United States, this designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended…
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