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North Carolina: Thousands of rare diseases don't have cures

Connor has Barth Syndrome; a rare disease that attacks just one in 400,000 with no known cure and shortened life expectancy.

Michelle-butt
General-manager
Nord
National-organization-for-rare-disorders
Barth-syndrome
Cure
Beichen
Medical
Medicine
O-cures
North-carolina

Maryland Stem Cell Research Fund Supports Innovative Women Researchers, Part 2 · BioBuzz

BioBuzz highlights more of the brilliant women scientists performing remarkable stem cell research with support from the MSCRF

Johns-hopkins-university
Maryland
United-states
University-of-maryland
Linda-resar
David-liu
Francis-collins
Hilary-vernon
Kan-cao
Department-of-cell-biology
Linkedin
Twitter

Stealth BioTherapeutics Granted Pre-NDA Meeting for Elamipretide as a Potential Treatment for Barth Syndrome

/PRNewswire/ Stealth BioTherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization.

Boston
Massachusetts
United-states
Adam-bero
Reenie-mccarthy
Exchange-commission
Us-food-drug-administration
Division-of-cardiology
Stealth-biotherapeutics-corp-nasdaq
Stealth-biotherapeutics-inc
Drug-administration
Chief-executive-officer

A mum's determination to find answers for her boy, and a family's wait for the day he can have a heart transplant

A mum's determination to find answers for her boy, and a family's wait for the day he can have a heart transplant
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New-zealand
Waikato
Australia
Richard-turpie
Kelsi-porter
Ministry-of-health
Canterbury-university
Chase-porter
Barth-syndrome
Waikato-hospital
Rainbow-place
High-cost-treatment-pool
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