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Editorial
. 2022 Jun;88(6):2480-2483.
doi: 10.1111/bcp.15350. Epub 2022 Apr 21.

Addressing the challenges of novel therapies in rare diseases with mechanistic perspectives: Missed opportunities or the way forward?

Janet Mifsud  1 , Noel Cranswick  2
Affiliations
Free article
Editorial

Addressing the challenges of novel therapies in rare diseases with mechanistic perspectives: Missed opportunities or the way forward?

Janet Mifsud et al. Br J Clin Pharmacol. 2022 Jun.
Free article
No abstract available

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References

REFERENCES

    1. Micallef J, Blin O. Orphan drug designation in Europe: a booster for the research and development of drugs in rare diseases. Therapie. 2020 Apr;75(2):133-139. doi:10.1016/j.therap.2020.02.003
    1. Kesselheim AS, Solomon DH. Incentives for drug development-the curious case of colchicine. N Engl J Med. 2010;362(22):2045-2047. doi:10.1056/NEJMp1003126
    1. Groft SC, Posada de la Paz M. Preparing for the future of rare diseases. Adv Exp Med Biol. 2017;1031:641-648. doi:10.1007/978-3-319-67144-4_34
    1. Forbes TA, Brown BD, Lai C. Therapeutic RNA interference: a novel approach to the treatment of primary hyperoxaluria. Br J Clin Pharmacol. 2022;88(6):2525-2538. doi:10.1111/bcp.14925
    1. Poddighe D, Romano M, Garcia-Bournissen F, Demirkaya E. Conventional and novel therapeutic options in children with familial Mediterranean fever: a rare autoinflammatory disease. Br J Clin Pharmacol. 2022;88(6):2484-2499. doi:10.1111/bcp.15149

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