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Case study, published in Nature Medicine, details improvements in nano-rare patient, Susannah’s seizure severity, neurologic function and quality of life
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n-Lorem received a $3 million donation to support its efforts in ASO research and discovery
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Parexel to provide clinical operations, real-world data solutions and expertise including medical and regulatory alongside innovative clinical research tools that will significantly enhance and streamline n-Lorem’s therapeutic development efforts
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World’s largest oligonucleotide manufacturer provides drug ingredients and financial donation to support development of experimental antisense medicines, helping n-Lorem reach more patients n-Lorem’s third partnership in the last quarter boosts resources to assist patients with ultra-rare mutations
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Global Genes launches RARE Health Equity Council to help build inclusive healthcare system and improve access for underserved rare disease patients.