FDA Cancer Office Taps Syapse for Real-world Evidence Development

NIH All of Us Project Tops 270,000 Sign-ups

Meet TRNDS 2019 Speaker Traci Schilling, PTC Therapeutics

Netflix's Medical Investigation Docuseries 'Diagnosis'

Ultragenyx Partners with GeneTx to Advance Treatment for Angelman Syndrome

Antisense Drugs for HD, ALS & Prion Could Meet the Need for Brain Treatments

'Dr. Google' Helps Some Patients Diagnose a Rare Disease

Evidation & Eli Lilly Study Uses Apple Devices, Apps to Predict Cognitive Impairment

HD Patients Prefer Less Invasive, No-Placebo Gene Therapy Trials

Meet TRNDS Speaker Tauna Batiste

C-Path and NORD Collaborate to Launch Rare Disease Data Platform

Neurogene Opens Natural History Study of CLN7, CLN5 Diseases at UT Southwestern

Rare Pediatric Disease PRVs: FDA Updates Guidance

Meet TRNDS 2019 Speaker PJ Brooks

(Podcast) Get to know Jayne Gershkowitz, Chief Patient Advocate at Amicus Therapeutics

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

NIH-Funded Project Aims to Build a ‘Google’ for Biomedical Data

Speeding Up Drug Discovery for Brain Diseases

NCATS Director Warns of 'Poorly Understood Public Health Implications of Rare Diseases'

Study refines ALS risk among first-degree relatives of patients with disease

NIH launches 5-year, $10 million study on acute flaccid myelitis

Neurogene Offers Access to Genetic Testing for Lysosomal Storage Disease

New Grants will Accelerate Clinical Trials in Rare Neurological Disorders

Gene Silencing Shows Promise for Treating Two Fatal Neurological Disorders