FAP News Today

August 4, 2020August 4, 2020

Tegsedi Eases Disease Progression in FAP Patients, Trial Data Show

FAP News Today

Treatment with Tegsedi (inotersen) slows neurological disease progression in people with familial amyloid polyneuropathy (FAP), results from a Phase 2/3 clinical trial show. The findings may be useful in designing ... Read more

July 21, 2020July 21, 2020

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

FAP News Today

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. The ... Read more

July 14, 2020July 14, 2020

New Nerve Imaging Tool May Aid Early FAP Diagnosis

FAP News Today

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 diagnosis ... Read more

July 9, 2020August 9, 2020

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

FAP News Today

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 in ... Read more

July 7, 2020July 8, 2020

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

FAP News Today

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 implicates ... Read more

June 23, 2020June 23, 2020

FAP Treatment Tegsedi Approved for Reimbursement in Italy

FAP News Today

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 1 ... Read more

June 22, 2020July 24, 2020

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

COVID-19 News, FAP News Today

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 support ... Read more

June 16, 2020June 16, 2020

Alnylam Gives $270K to Support Projects by 7 FAP Advocacy Groups

FAP News Today

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

June 10, 2020July 11, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

COVID-19 News, FAP News Today

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 for ... Read more

June 9, 2020June 10, 2020

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

FAP News Today

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 positive ... Read more

June 2, 2020June 2, 2020

Tegsedi Approved for Reimbursement in Portugal

FAP News Today

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, 2020June 29, 2020

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

COVID-19 News, FAP News Today

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 therapies ... Read more

May 26, 2020May 26, 2020

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

FAP News Today

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 seen ... Read more

May 25, 2020June 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

FAP News Today

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was ... Read more

May 21, 2020June 22, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

COVID-19 News, FAP News Today

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, ... Read more