News Non-Val30Met FAP Patients Exhibit Cardiac and Blood Vessel Dysfunction, Study Shows

News Tegsedi Found to Improve Neuropathy Symptoms, Quality of Life in FAP Patients in Phase 3 Trial

News Tegsedi Approved in EU to Treat FAP Patients with Stage 1 or 2 Polyneuropathy

News Phase 3 Trial Results Supporting Benefits of Patisiran in FAP Published in NEJM

News Rare Case of Cardiac Symptoms, Fluid Accumulation Reported in Portuguese FAP Patient

News Vyndaqel, Liver Transplant Equally Effective at Preventing FAP Progression, Study Shows

News Unique Set of Predictors to Distinguish Familial Amyloid Polyneuropathy Identified in Analysis

News Rare Case: Upper Limb Symptoms Preceded FAP Val30Met Mutation Diagnosis

News Noninvasive Sudoscan May Help Assess Severity of FAP in Patients, Study Suggests

Just Published, News Eye Blood Vessel Alterations Common in FAP with Val30Met Mutation, Study Finds

News US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

News EU’s CHMP Recommends Approval of Tegsedi (Inotersen) for Treatment of FAP

News #ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

News Vyndaqel Fails to Prevent Progression of Rare FAP Variant, Case Report Shows

News ‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News #ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News Rare Mutation Identified in Libyan Patient with Late-onset FAP, Case Study Reports

News #ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12

News Vyndaqel Slows Nerve Cell Damage, Improves Quality of Life of FAP Patients, Real-World Study Shows

News Protein Interaction and Aggregation Studies May Provide Key Data for Treatment of Amyloid Diseases, Study Says

News Akcea Completes Deal That Gives It Marketing Rights to FAP Treatment Inotersen

News #AAN2018 – FAP Linked to Serious Heart Rate Control Abnormalities, Study Shows

News NIH Agency Pioneers Collaborative Research into Rare Diseases

News Japanese Region Has Several Examples of Rare FAP Mutation, Case Study Reports

News Combination of Three Tests Can Spot TTR-FAP Early, Study Reports

News Psychological Support May Benefit Certain People at Risk for FAP, Study Suggests

News Inotersen Clinical Development Update Presented at Conference

News Ionis and Akcea Partner to Market FAP Therapy Candidate Inotersen

News Genome Sequencing and Its Clinical Potential Focus of NYC Rare Disease Day Event

News Standardized Measures Needed to Assess Autonomic Involvement in FAP, Study Suggests

News ‘A Disease May Be Rare, Hope Should Never Be,’ Says Boy with SMARD at Connecticut Rare Disease Day Event

News Imaging Agent Can Detect Early Cardiac Involvement in FAP Patients, Study Finds

News Rare Disease Groups Welcome FDA’s Embrace of ‘Real World’ Data in Clinical Trials

News US Lawmakers Urge Bipartisan Support for Rare Disease Research, Patients’ Needs

News Retrophin, Horizon Donate $3M Each to Rare Disease Institute at Children’s National

News BioNews to Cover 3 Rare Disease Day Events, Including NIH Conference

News Shire, Microsoft and EURORDIS Partner to Shorten Multi-year Diagnosis of Rare Diseases

News Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28

News Taiwanese Team Develops Mouse Model That Displays the Early Stages of Human FAP

News Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress

News Changes in Walking Patterns Could Help Predict FAP, Portuguese Study Reports

News NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases

News Japanese Experts Develop Guidelines for the Country’s Unique TTR-FAP Variations

News Vyndaqel May Not Protect FAP Patients Against Anemia, Study Finds

News Alnylam Continuing FAP Treatment Development Without Former Partner Sanofi

News Inotersen a Step Closer to Approval as FDA Grants Priority Review to Ionis’ NDA

News Surgical Joint Fusion Is Effective Treatment for Rare Knee Deformity in FAP, Case Report Shows

News Researchers Come Up with Tool Kit of Tests for Early Diagnosis of TTR-FAP

News Prevalence Study Finds FAP May Be More Common Worldwide Than Previously Thought

News Alnylam and Sanofi Ask European Regulators to Approve Patisiran for FAP

News Complete New Drug Application for FAP Therapy Patisiran Submitted to FDA

News FDA Expands Patisiran’s Orphan Drug Status to Another Form of Familial Amyloidosis

News Cognitive Impairment Common Among Older, Untreated FAP Patients

News MMP-14 Levels May Serve as Biomarker and Therapeutic Target in FAP Patients

News Alnylam Starts Rolling Submission of Application Asking FDA to Approve Patisiran

News Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US

News Vyndaqel Can Slow Nerve-cell Damage in FAP, Regardless of Gene Mutation, Analysis Finds

News EMA Regulatory Committee Grants Accelerated Assessment to Patisiran to Treat FAP

News Inotersen, Potential FAP Treatment, Under Review for Approval in Europe

News Alnylam Set to Seek Regulatory Approval for Patisiran With Robust Findings in Phase 3 FAP Trial

News Children’s National, NORD Partner to Create Rare Disease ‘Centers of Excellence’

News FAP Patients Have a Moderate Chance of Having Unaffected Children, Study Suggests

News Psychological, Psychiatric Support Can Be Beneficial for TTR-FAP Patients, Study Suggests

News Portuguese Study Links Extra Copies of Mitochondrial DNA to Earlier Development of FAP

News #NORDsummit – Alternatives to Large Placebo Trials, Grant Awards Among Ways FDA Supporting Rare Diseases, Chief Says

News Latest NEURO-TTR Trial Data Confirm Inotersen’s Potential to Treat FAP Patients

News #NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says

News UMass Receives $10M Donation to Launch Rare Diseases Research Institute

News Late-onset FAP Patients Commonly Show Involuntary Nerve and Heart Abnormalities, Study Finds

News Portuguese Man with FAP-related Glaucoma Treated Without Surgery, Case Study Shows

News Serum Proteins May Be Used to Aid Diagnosis and Prognosis of FAP, Study Finds

News Case Study: FAP Liver Caused Heart Amyloidosis in Recipient After Transplant

News FAP Patients Might Have New Treatment Before Year’s End, as Trial Data Show Patisiran’s Benefits

News Study Explains Phase 3 Clinical Trial Evaluating Alnylam’s Patisiran as Potential FAP Therapy

News Imaging Is as Good as Tissue Sampling at Detecting Amyloid Deposits in Some Organs, Study Finds

News Symptoms of Autonomic Neuropathy May Suggest Diagnosis of Primary Amyloidosis

News Tafamidis Delays Disease Progression in Patients with Transthyretin Amyloid Polyneuropathy, Pfizer Study Suggests

News Disease Knowledge in People With FAP Family History Differs From Those With Treatable Diseases

News Rare TTR Mutation Seen to Cause FAP in Chinese Family

News Alnylam’s Patisiran Shows Promise for Treating FAP in Phase 2 Trial

News Ionis Continues FAP Therapy Development Alone as GSK Reconsiders Rare Diseases Field

News TRAM Trial to Identify Prevalence of Ttr Gene Errors in German Study of FAP

News MIBG Scans Prove Valuable in Determining Familial Amyloid Polyneuropathy Patients’ Outcomes, Study Shows

News 6 Genes Seen to Influence Age at Which Common Type of FAP Strikes

News Doctors Report Case of Late-Onset FAP With Difficult Diagnosis

News Mutated TTR Protein Triggers Inflammation in FAP Patients and Carriers, Study Finds

News Magnetic Resonance Neurography Can Detect TTR-FAP Nerve Lesions, Study Reports

News Amyloid Deposits in Skin Samples Could Be Alternative to Biopsy in Diagnosing FAP, Study Suggests

News Inotersen Seen to Improve Neurological Symptoms, Quality of Life of FAP Patients in Phase 3 Trial

News DNA Mutation May Play a Role in Age at Which Familial Amyloid Polyneuropathy Starts, Study Reports