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November 12, 2019November 12, 2019

Protein Modification May Lead to Heart Problems in FAP by Promoting Aggregation of Mutant TTR, Study Says

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The addition of the amino acid homocysteine to the mutated transthyretin (TTR) protein found in patients with familial amyloid polyneuropathy may contribute to heart problems in these patients, a ... Read more

November 5, 2019November 5, 2019

Study Describes Characteristics of Autonomic Symptoms in Hereditary Amyloidosis

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Autonomic symptoms are common among patients with hereditary ATTR amyloidosis, usually accompany the progression of peripheral neuropathy, and are more prevalent in carriers of Val30Met mutations, a review of ... Read more

October 30, 2019October 30, 2019

Early Childhood Onset of FAP Described in Korean Teenager

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The case of a 17-year-old boy who experienced the first symptoms of familial amyloid polyneuropathy (FAP) before the age of 3, calls attention to the possibility of pediatric cases ... Read more

October 23, 2019October 23, 2019

Vyndaqel Likely to Treat FAP Linked to Mutation Common in Asians, Lab Study Suggests

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Vyndaqel (tafamidis) increases the stability of the transthyretin protein and may help people with familial amyloidosis polyneuropathy (FAP) carrying a TTR gene mutation common in Asian populations, an early study from Taiwan suggests. ... Read more

October 18, 2019October 18, 2019

Gastroenterologists Should Be Part of Multidisciplinary Team to Treat FAP, Study Suggests

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As early detection and management of gastrointestinal (GI) disturbances is key in familial amyloid polyneuropathy (FAP), gastroenterologists should be part of a multidisciplinary team to treat these patients, a ... Read more

October 8, 2019October 8, 2019

Noninvasive Imaging Techniques May Help Identify, Monitor Eye Problems in FAP, Study Shows

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Noninvasive imaging techniques may be helpful for identifying and monitoring eye problems in familial amyloid polyneuropathy (FAP), a study shows. Titled “Multimodal retinal imaging of familial amyloid polyneuropathy,” the ... Read more

October 1, 2019October 1, 2019

Study Supports Genetic Testing for FAP in Neuropathy with No Known Cause

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Genetic sequencing can help to diagnose familial amyloid polyneruopathy (FAP) in people diagnosed with neuropathy of unknown origin — and sequencing is more reliable than biopsies for diagnosis, the results ... Read more

September 27, 2019September 27, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

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Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more

September 24, 2019September 24, 2019

Urinary and Sexual Dysfunction Prevalent in FAP Patients, Review Finds

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Urinary and sexual problems are highly prevalent in people with familial amyloid polyneuropathy (FAP) and should be tackled by a comprehensive and multidisciplinary management plan to prevent further complications ... Read more

September 17, 2019September 17, 2019

AKCEA-TTR-LRx Reduces Levels of TTR in Healthy Volunteers, Phase 1 Trial Shows

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Akcea Therapeutics’ next-generation antisense medicine AKCEA-TTR-LRx (ION-682884) — currently in development for the treatment of all forms of familial amyloid polyneuropathy (FAP) — reduces the levels of a key ... Read more

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