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New Grants will Accelerate Clinical Trials in Rare Neurological Disorders
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Neurogene Offers Access to Genetic Testing for Lysosomal Storage Disease
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NIH launches 5-year, $10 million study on acute flaccid myelitis
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Study refines ALS risk among first-degree relatives of patients with disease
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NCATS Director Warns of 'Poorly Understood Public Health Implications of Rare Diseases'
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Speeding Up Drug Discovery for Brain Diseases
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NIH-Funded Project Aims to Build a ‘Google’ for Biomedical Data
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Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
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(Podcast) Get to know Jayne Gershkowitz, Chief Patient Advocate at Amicus Therapeutics
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Meet TRNDS 2019 Speaker PJ Brooks
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Rare Pediatric Disease PRVs: FDA Updates Guidance
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Neurogene Opens Natural History Study of CLN7, CLN5 Diseases at UT Southwestern
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C-Path and NORD Collaborate to Launch Rare Disease Data Platform
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Meet TRNDS Speaker Tauna Batiste
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HD Patients Prefer Less Invasive, No-Placebo Gene Therapy Trials
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Evidation & Eli Lilly Study Uses Apple Devices, Apps to Predict Cognitive Impairment
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'Dr. Google' Helps Some Patients Diagnose a Rare Disease
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Antisense Drugs for HD, ALS & Prion Could Meet the Need for Brain Treatments
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Ultragenyx Partners with GeneTx to Advance Treatment for Angelman Syndrome
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Netflix's Medical Investigation Docuseries 'Diagnosis'
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NIH All of Us Project Tops 270,000 Sign-ups
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FDA Cancer Office Taps Syapse for Real-world Evidence Development
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Gene Linked to Rare Neurological Disorder May Play a Role in Alzheimer’s
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Gene Therapy Developers Issue Principles for Human Genome Editing
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Forget Single Genes: CRISPR Now Cuts & Splices Whole Chromosomes
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TRNDS Speaker Benjamin Schlatka, MC10
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TRNDS Speaker Danielle Edwards, Jett Foundation
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His daughter has a rare disorder. He’s developing a novel gene therapy.
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A Doctor Tested a New Treatment on Himself, Now it's Saving Other Lives
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Study Presents Clinical, Genetic Landscape of Pompe Disease
Jeanne Paiko
Center Administrative Manager, CHeT