Video tag: Rare Disease

Patient Data builds the Preclinical Toolbox for the CACA1A Foundation
Boy with SPG50, an Ultra-Rare Disease begins Groundbreaking Gene Therapy Clinical Trial
Pfizer on Gene Therapy and AAV Vectors for Rare Disease
n-Lorem Foundation Provides Experimental Medicine to Child With Ultra Rare Disease KIF1A
Principal Investigator from Leiden Discusses the Role of Seed Funding for Research on Langerhans Cell Histiocytosis
San Antonio Texas Firefighter Participates in Clinical Trials for Stiff Person Syndrome
Bruno Boulanger Head of Statistics at PharmaLex Discusses Bayesian Statistics and Rare Disease Clinical Trials
European Joint Program on Rare Diseases Builds Rare Disease Research Ecosystem
Sangamo Therapeutics Executive Discusses Clinical Trials of Fabry Disease
Drug Repurposing for Rare Disease from RareBeacon
Cannonball Run to Raise Funds for SYNGAP1 Clinical Trials
Men With Rare Disease Talk About Mental Health
Fundraiser for Clinical Studies of KCNC1
1st Annual Scramble for SYNGAP1 Clinical Trials
FSHD Clinical Trials in Australia
Rare Disease SynGAP Clinical Trials
Clinical trial for Rare Disease SLC6A1
Groundbreaking Epidermolysis Bullosa Clinical Trials thanks to Venture Philanthropy
Gigantic Donation to Cure FSHD
Ultra Rare Disease Awareness Enhanced through Fundraiser
IRF2BPL Gene Foundation Established.
A New Foundation to Help Patients with Ultra Rare Disease
IQVIA Looks at AI and Machine Learning in Finding Patients for Clinical Trials
Rare Disease PMD Clinical Trials
n-Lorem Delivers HOPE to Treat Ultra-rare Diseases
An Industry Look at Clinical Trials For Rare Diseases
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