FAP News Today

July 28, 2021July 28, 2021

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

FAP News Today

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other ... Read more

July 26, 2021July 26, 2021

Depression, Anxiety Common With FAP, Patient Survey Finds

FAP News Today

Challenges with mental health often follow a person being told that they carry a mutation that causes familial amyloid polyneuropathy (FAP), a rare and progressive disease of adulthood, a study ... Read more

July 23, 2021July 23, 2021

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

FAP News Today

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies ... Read more

July 19, 2021July 19, 2021

Novo Nordisk Acquires Prothena’s PRX004, Plus Full ATTR Program

FAP News Today

Novo Nordisk has acquired Prothena’s clinical-stage antibody PRX004, an investigational therapy for people with hereditary transthyretin amyloidosis (ATTR), which also includes familial amyloid polyneuropathy (FAP), as well as other non-hereditary ... Read more

July 15, 2021July 15, 2021

Nominations Open for 2022 Eurordis Black Pearl Awards

FAP News Today

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work ... Read more

July 12, 2021July 12, 2021

Disease-modifying Therapies Found to Extend Patient Survival

FAP News Today

Treatment with disease-modifying therapies can extend survival in people with familial amyloid polyneuropathy (FAP), a new study indicates. The study, “Use of Drugs for ATTRv Amyloidosis in the Real World: ... Read more

June 30, 2021June 30, 2021

Centogene Sets Mission to Cure 100 Rare Diseases in Next 10 Years

FAP News Today

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, ... Read more

June 30, 2021June 30, 2021

Early Data Favorable for NTLA-2001 Gene-editing Therapy

FAP News Today

A single dose of Intellia Therapeutics’ investigational CRISPR/Cas9-based gene-editing therapy NTLA-2001 safely results in a rapid and strong reduction in the levels of the damaging transthyretin (TTR) protein, according to ... Read more

June 28, 2021June 28, 2021

FDA to Decide on Vutrisiran’s Possible Approval by April 2022

FAP News Today

Alnylam Pharmaceuticals’ application for the approval of vutrisiran, its investigational therapy for familial amyloid polyneuropathy (FAP), is being reviewed by the U.S. Food and Drug Administration (FDA), with a decision ... Read more

June 21, 2021June 21, 2021

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

FAP News Today

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health ... Read more

June 16, 2021June 16, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

COVID-19 News, FAP News Today

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the ... Read more

June 14, 2021June 14, 2021

Amyl Raises Funds to Develop Therapies for Amyloidosis

FAP News Today

Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance therapeutic candidates for familial amyloid polyneuropathy (FAP) and other forms of ... Read more

June 7, 2021June 7, 2021

Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases

FAP News Today

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in ... Read more

June 7, 2021June 7, 2021

APOLLO-B Trial of Onpattro Fully Enrolls Patients with Heart Disease

FAP News Today

Enrollment is now completed in the Phase 3 APOLLO-B trial, which is assessing the safety and efficacy of Onpattro (patisiran) to treat heart disease in people with transthyretin-mediated (ATTR) amyloidosis, ... Read more

June 2, 2021June 2, 2021

Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill

FAP News Today

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in ... Read more