Benzbromarone (BBM), a medicine used to treat gout, can stabilize the TTR protein to the same degree as therapies used…
News
The case of a woman of Afro-Caribbean descent with early onset transthyretin (TTR) amyloidosis, who was found to carry a TTR mutation more commonly seen…
PRX004, an investigational treatment by Prothena, can reduce the progression of nerve damage (neuropathy) and improve heart function in people with hereditary transthyretin amyloidosis (ATTR),…
Abdominal fat pad biopsy is an accurate diagnostic tool for patients with suspected hereditary transthyretin amyloidosis…
To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the…
Onpattro (patisiran) results in sustained improvements in polyneuropathy, quality of life, and other symptoms among people with familial amyloid polyneuropathy (FAP), while showing…
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their…
Onpattro (patisiran) is more effective than Tegsedi (inotersen) at reducing neurological impairment and improving quality of…
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient…
Three recently approved therapies for treating familial amyloid polyneuropathy (FAP), also known as hereditary transthyretin amyloidosis with polyneuropathy, have won their developers Best…
Recent Posts
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- Women with ATTR-CM face gaps in treatment, new study reports