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December 6, 2019December 6, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

December 3, 2019December 3, 2019

Alnylam Opens ‘Advocacy for Impact’ Grant Program Supporting FAP, Like Patient Groups

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Alnylam Pharmaceuticals has opened its second annual competitive grants program, supporting patient advocacy projects worldwide that aim to address the unmet and diverse needs of ATTR amyloidosis, a group of ... Read more

November 26, 2019November 26, 2019

Akcea and Ionis Launch Phase 3 Trial of AKCEA-TTR-LRx in People With FAP

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Akcea Therapeutics and Ionis Pharmaceuticals announced the launch of a Phase 3 clinical trial evaluating the safety and efficacy of AKCEA-TTR-LRx (ION-682884) for the treatment of adults with familial ... Read more

November 19, 2019November 19, 2019

Gait Tracking Using RGB-D Camera Could Aid Doctors in Diagnosing, Managing FAP, Study Shows

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An affordable, portable, and non-invasive human motion tracking system based on an RGB-D camera can be useful for monitoring gait impairments in people with familial amyloid polyneuropathy (FAP), a ... Read more

November 15, 2019November 16, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more

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

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