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July 21, 2020July 21, 2020

Long-term Onpattro Use Safely Halts FAP Progression, Phase 2 Data Show

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Long-term treatment with Onpattro (patisiran) safely and effectively halts or reverses disease progression in people with familial amyloid polyneuropathy (FAP), final data from a two-year Phase 2 trial show. ... Read more

July 14, 2020July 14, 2020

New Nerve Imaging Tool May Aid Early FAP Diagnosis, Study Suggests

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Magnetization transfer ratio (MTR), an MRI scanning tool based on nerve imaging, may help diagnose familial amyloid polyneuropathy (FAP) in its early stages, a study suggests. With delays in ... Read more

July 9, 2020July 9, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

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The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease ... Read more

July 7, 2020July 7, 2020

Calprotectin Protein Could Be Non-invasive Biomarker for FAP, Study Suggests

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Levels of calprotectin, a marker of inflammation, are elevated in the feces of people with familial amyloid polyneuropathy (FAP) who experience gastrointestinal (GI) symptoms, a study suggests. This finding ... Read more

June 23, 2020June 23, 2020

FAP Treatment Tegsedi Approved for Reimbursement in Italy

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The Italian Medicines Agency has added Tegsedi (inotersen), a treatment for familial amyloid polyneuropathy (FAP), to the list of reimbursed therapies in the country. Tegsedi was approved to treat stage ... Read more

June 22, 2020July 28, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

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The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 16, 2020June 17, 2020

Alnylam Gives $270K to Support Projects by 5 FAP Advocacy Groups

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Five advocacy groups for familial amyloid polyneuropathy (FAP) are sharing a total $270,000 award given by Alnylam Pharmaceuticals to support projects looking to promote patient care, and greater disease ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

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The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis ... Read more

June 9, 2020June 9, 2020

Tegsedi Shows Sustained Benefits, Long-term Safety in FAP Patients

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Tegsedi (inotersen) continued to slow disease progression and improve the quality of life in people with familial amyloid polyneuropathy (FAP) after two years of treatment, and also maintained a ... Read more

June 2, 2020June 2, 2020

Tegsedi Approved for Reimbursement in Portugal

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The Portuguese Medicine Regulatory Authority, known as Infarmed, has added Tegsedi (inotersen) — an approved treatment for stage 1 or 2 polyneuropathy in adults with familial amyloid polyneuropathy (FAP) ... Read more

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