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Rare Trait Swiss is a Non-Profit organization which contribute to raise awareness of an Lysosomal rare disease called AGU. Our goal is to fund the medical research to manufacture the cure for AGU kids and to fund a clinical trial using gene therapy
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Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.
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The Lily Foundation is a UK charity dedicated to fighting mitochondrial disease. We work in partnership with the NHS and leading mitochondrial clinics to support those affected by the condition, raise awareness, and fund research into effective treatments and therapies. Our long-term goal is to find a cure for mitochondrial disease.
Candle in the dark
Candle in the Dark is a charitable organisation funding research into gene therapy for inherited eye disorders causing blindness in young children. Its aim is to help develop effective treatments, ultimately providing a cure for blindness and visual impairment. Visit our pages to find out more and see how you can help.
Candle in the dark
Candle in the Dark is a charitable organisation funding research into gene therapy for inherited eye disorders causing blindness in young children. Its aim is to help develop effective treatments, ultimately providing a cure for blindness and visual impairment. Visit our pages to find out more and see how you can help.
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