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  2. CureCMT4J | Advancing Treatments for Rare Diseases

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  4. Allies in Rare Disease - Global Genes
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  6. Allies in Rare Disease - Global Genes
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  8. Gene and cell therapies to counteract neuroretinal diseases
    Find here information on eye disease research and the priority program SPP 2127: Gene and cell based therapies to counteract neuroretinal degeneration.

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  14. Cycle Pharmaceuticals – Treatments for rare disease patients
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  22. Gene Therapy for Rare Disorders Europe 2021 | Home
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  28. ACPGBI | Advancing knowledge and treatment of bowel disease
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  36. Cambridge Rare Disease Network - Cambridge Rare Disease Network
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  39. everylifefoundation.org
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  52. 4th Annual Gene Therapy for Rare Disorders 2021 | Home
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  54. Hypophosphatasia Causes, Symptoms and Treatment | Rare Bone Disease | HPP
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  60. MCDS-Therapy | EU Horizon 2020 project | Rare bone disease
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  78. IORD - Indian Organization for Rare Diseases | A not-for-profit organization for Rare Diseases in India.

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  84. Journal of Rare Cardiovascular Diseases
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  85. ultragenyx.com
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  86. Rare Genetic Diseases | Ultragenyx
    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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  88. HRA Pharma Rare Diseases
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  94. Radico - Rare Disease Cohorts
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  96. Home - Rare Disease UK
    What's the UK Rare Diseases Strategy? Where can I find information & support? How can I fundraise for Rare Disease UK? The latest news from RARE DISEASE UK... Lauren’s Story Living with Hereditary Amyloidosis One in a Million Abigail’s story Will’s Story Our work We work on ...

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  100. Disease Treatment | ReKNEW
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  108. Rare diseases Luxembourg , we care 4 rare! - ALAN
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  112. Home • ATTC Network - Advanced Therapy Treatment Centre
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  124. BioCentriq | Cell & Gene Therapies
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