Spotlight
Mar 01, 2024
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Hereditary transthyretin (hATTR) amyloidosis, also known as variant ATTR (ATTRv) amyloidosis, is a rare disease affecting about 50,000 people worldwide. It's caused…
Individuals suffering with rare diseases had very few therapeutic options 40 years ago. But that began to change with the stroke of a pen by then-President Ronald…
UC San Diego Health is first in the nation to offer a new injectable medication to patients with nerve damage caused by hereditary transthyretin amyloidosis, a rare…
AL amyloidosis is a life-threatening disease related to bone marrow cancer, multiple myeloma. It is caused when a person's antibody-producing white blood cells (i.e…
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration (FDA) has extended the review timeline of…
Investigators have found that intravenous infusion of a CRISPR-Cas9 based gene therapy decreases serum transthyretin (TTR) protein in patients with hereditary TTR…
AL Amyloidosis and Agent Orange
Veterans who develop AL amyloidosis and were exposed to Agent Orange or other herbicides during military service do not have to…
The Veterans Health Administration exists to integrate health care services for those who served in the U.S. Armed Forces. Yet for many veterans, getting access to…