A new publication about incorporating Spinal Muscular Atrophy (SMA) in newborn screening programs across Europe is now available in the Rare Disease and Orphan Drugs Journal. The paper highlights how patient advocacy organizations represent a key partner in ensuring equitable and early diagnosis. đź“– Read the full publication here: https://lnkd.in/emYepUDXđź“š More publications on Newborn Screening: […]
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The latest IRDiRC paper, “Drug Repurposing for Rare: Progress and Opportunities for the Rare Disease Community” has been published in Frontiers in Medicine. The paper is available here. This open-access publication, reflecting the collaborative efforts of the IRDiRC Drug Repurposing Task Force and the IRDiRC Therapies Scientific Committee over the past 1.5 years, is a […]
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New Publication: Making Rare Disease Research Attractive to Companies by IRDiRC Chrysalis Task Force
Accessible at https://doi.org/10.1177/26330040231188979, the Chrysalis Task Force identified key financial and non-financial factors that make rare disease research and development attractive to companies. More information on the Chrysalis Task Force here: https://irdirc.org/chrysalis-project/ « It was a real pleasure working with such a talented IRDiRC Task Force. We also truly appreciate the critical input provided by the […]
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