Study Presents Clinical, Genetic Landscape of Pompe Disease

A Doctor Tested a New Treatment on Himself, Now it's Saving Other Lives

His daughter has a rare disorder. He’s developing a novel gene therapy.

TRNDS Speaker Danielle Edwards, Jett Foundation

TRNDS Speaker Benjamin Schlatka, MC10

Forget Single Genes: CRISPR Now Cuts & Splices Whole Chromosomes

Gene Therapy Developers Issue Principles for Human Genome Editing

TRNDS Speaker Nancy Yu, RDMD

TRNDS Speaker Rachel McMinn, Neurogene

Gene Linked to Rare Neurological Disorder May Play a Role in Alzheimer’s

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