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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

September 10, 2019September 10, 2019

Alexion Obtains Japanese Rights to Potential FAP Therapy AG10; Phase 3 Trial Planned

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Alexion Pharmaceuticals has obtained exclusive rights in Japan to develop and market AG10, an oral treatment candidate for familial amyloid polyneuropathy (FAP), also called ATTR polyneuropathy (ATTR-PN). AG10 is a small ... Read more

September 2, 2019September 2, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 29, 2019September 9, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

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Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 27, 2019August 27, 2019

Lower Cathepsin E Levels Could Underlie Impaired Function of Macrophages in FAP, Study Suggests

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The lower levels of a protein called cathepsin E, upon activation of a type of white blood cells called macrophages, in mice with familial amyloid polyneuropathy (FAP) suggest a ... Read more

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