Spotlight
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…
The Amyloidosis Allianceexternal link, opens in a new tab and worldwide patients organization has created an International Day of Awareness dedicated to this rare…
The Amyloidosis Allianceexternal link, opens in a new tab and worldwide patients organization has created an International Day of Awareness dedicated to this rare…
Is the COVID-19 vaccine safe?
How effective is it? The United States Food and Drug Administration (FDA) has granted emergency approval for the COVID-19 vaccine.…
Human bodies constantly produce thousands of perfectly folded proteins, but some proteins get misfolded. An excess of these misfolded proteins can overwhelm the…
At this time of crisis and with the health, safety and well-being of patients and caregivers as its top priorities, the National Organization for Rare Disorders (…