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July 9, 2020July 9, 2020

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

News, syndicated

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

May 29, 2020May 29, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

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While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene ... Read more

May 26, 2020May 26, 2020

Onpattro Treatment Improves Quality of Life in FAP Patients, Study Shows

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Treatment with Onpattro (patisiran) improves multiple aspects of quality of life in people with familial amyloid polyneuropathy (FAP), findings from a Phase 3 clinical trial show. The rapid deterioration ... Read more

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