FAP News Today

October 18, 2021October 19, 2021

Tegsedi Gets Innovative Medication Category Pricing in Brazil

FAP News Today

Tegsedi (inotersen), an approved treatment for familial amyloid polyneuropathy (FAP), has received a Category 1 classification by the legal body tasked with economic regulation of medications in Brazil. Category 1 ... Read more

October 15, 2021October 15, 2021

Rare Disease Diversity Coalition Awards $600K to Combat Disparities

COVID-19 News, FAP News Today

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC ... Read more

October 11, 2021October 11, 2021

Onpattro Now Will Be Reimbursed for Most Patients in Canada

FAP News Today

Onpattro (patisiran) is now reimbursed for patients with familial amyloid polyneuropathy (FAP) living across most regions of Canada, according to Alnylam Pharmaceuticals, the therapy’s developer. The announcement comes following a ... Read more

October 4, 2021October 4, 2021

‘Rare’ Documentary in Kickstarter Campaign to Raise $45K by Oct. 28

FAP News Today

A crowdfunding campaign aiming to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and ... Read more

September 30, 2021September 30, 2021

New Data-sharing Program Aims to Speed Innovation in Rare Diseases

FAP News Today

A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and ... Read more

September 27, 2021September 28, 2021

Attralus Gets $116M Financing Boost to Develop Imaging Agent

FAP News Today

Attralus has raised $116 million to support the clinical development of AT-01, its diagnostic imaging agent for systemic amyloidosis, a group of rare conditions that includes familial amyloid polyneuropathy (FAP). Proceeds ... Read more

September 23, 2021September 23, 2021

Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness

COVID-19 News, FAP News Today

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games ... Read more

September 20, 2021September 20, 2021

Vutrisiran Now Under Review for Approval in Europe for FAP Treatment

FAP News Today

The European Medicines Agency (EMA) has agreed to review Alnylam Pharmaceuticals’ application requesting the approval of vutrisiran, its second-generation RNA interference (RNAi) therapy candidate for familial amyloid polyneuropathy (FAP). Hereditary ATTR ... Read more

September 17, 2021September 17, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

FAP News Today

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 13, 2021September 13, 2021

Further Benefits With Vutrisiran Seen in FAP Patients in Phase 3 Trial

FAP News Today

Vutrisiran, an investigational therapy for familial amyloid polyneuropathy (FAP), eased neurological impairments, lessened heart stress, and ameliorated quality of life, nutritional status, and social engagement in adult patients, according to ... Read more

September 9, 2021September 9, 2021

Group Focuses on Rare Disease Clinical Trial Participation

FAP News Today

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

September 3, 2021September 3, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

FAP News Today

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more

August 30, 2021August 31, 2021

Tegsedi Helps Preserve Patient Quality of Life in Extension Study

FAP News Today

Long-term treatment with Tegsedi (inotersen) helps preserve quality of life in people with familial amyloid polyneuropathy (FAP) for up to three years, according to data from the Phase 3 NEURO-TTR ... Read more

August 26, 2021August 26, 2021

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

FAP News Today

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The ... Read more

August 23, 2021August 23, 2021

Eye Abnormalities May Serve as Early Biomarkers for hATTR Amyloidosis

FAP News Today

Changes in the function of the retina, in combination with nerve damage in the cornea, can be used as early biomarkers of hereditary transthyretin (hATTR) amyloidosis, also known as familial ... Read more