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Rare Disease
Usher Syndrome Coalition Advocated for Usher Syndrome Research Funding Using Ush Champions and More
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Usher Syndrome
Rare Disease
Patient Data builds the Preclinical Toolbox for the CACA1A Foundation
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Rare Disease
Data Science
CACNA1A
Boy with SPG50, an Ultra-Rare Disease begins Groundbreaking Gene Therapy Clinical Trial
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SPG50
Rare Disease
Fundraising
Pfizer on Gene Therapy and AAV Vectors for Rare Disease
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Rare Disease
Gene Therapy
n-Lorem Foundation Provides Experimental Medicine to Child With Ultra Rare Disease KIF1A
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Rare Disease
KIF1A
Fundraising
Principal Investigator from Leiden Discusses the Role of Seed Funding for Research on Langerhans Cell Histiocytosis
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Rare Disease
Histiocytosis
Fundraising
San Antonio Texas Firefighter Participates in Clinical Trials for Stiff Person Syndrome
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Rare Disease
Bruno Boulanger Head of Statistics at PharmaLex Discusses Bayesian Statistics and Rare Disease Clinical Trials
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Rare Disease
Data Science
European Joint Program on Rare Diseases Builds Rare Disease Research Ecosystem
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Rare Disease
Clinical Trials
Sangamo Therapeutics Executive Discusses Clinical Trials of Fabry Disease
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Rare Disease
Fabry
Drug Repurposing for Rare Disease from RareBeacon
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Rare Disease
Fundraising
AI
Cannonball Run to Raise Funds for SYNGAP1 Clinical Trials
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SynGAP
Rare Disease
Men With Rare Disease Talk About Mental Health
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Rare Disease
Mental Health
Fundraiser for Clinical Studies of KCNC1
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Rare Disease
KCNC1
Fundraising
1st Annual Scramble for SYNGAP1 Clinical Trials
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SynGAP
Rare Disease
Fundraising
FSHD Clinical Trials in Australia
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Rare Disease
Fundraising
FHSD
Rare Disease SynGAP Clinical Trials
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SynGAP
Rare Disease
Clinical trial for Rare Disease SLC6A1
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SLC6A1
Rare Disease
Groundbreaking Epidermolysis Bullosa Clinical Trials thanks to Venture Philanthropy
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Rare Disease
Gigantic Donation to Cure FSHD
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Rare Disease
Ultra Rare Disease Awareness Enhanced through Fundraiser
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Rare Disease
IRF2BPL Gene Foundation Established.
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Rare Disease
IRF2BPL
A New Foundation to Help Patients with Ultra Rare Disease
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Rare Disease
IQVIA Looks at AI and Machine Learning in Finding Patients for Clinical Trials
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Rare Disease
AI
Rare Disease PMD Clinical Trials
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Rare Disease
Fundraising
n-Lorem Delivers HOPE to Treat Ultra-rare Diseases
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Rare Disease
An Industry Look at Clinical Trials For Rare Diseases
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Rare Disease
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