The UN Convention on the Rights of the Child and other Human rights instruments, recognising that children are rights-holders with a progressively evolving ability to make their own decisions, endorsed changes in the general perception of the autonomy and protection of children regarding their capacity to participate in decision-making. However, on matters concerning their health, […]
Read MoreMedics4RareDiseases has launched its online, interactive e-learning platform dedicated to teaching medics the fundamentals of rare disease and help them manage both their undiagnosed and diagnosed patients. Sign up here to access M4RD courses
Read MoreMedics4RareDiseases has launched its online, interactive e-learning platform dedicated to teaching medics the fundamentals of rare disease and help them manage both their undiagnosed and diagnosed patients. Sign up here to access M4RD courses
Read MoreHeritable human genome editing has been proposed as a means of helping parents avoid passing genetic diseases to future generations. But can heritable human genome editing be used safely? On February 26, 2021 at 9:00 am EST (2 pm GMT / 3pm CET / 10 pm CST), join members of the International Commission on the […]
Read MoreHeritable human genome editing has been proposed as a means of helping parents avoid passing genetic diseases to future generations. But can heritable human genome editing be used safely? On February 26, 2021 at 9:00 am EST (2 pm GMT / 3pm CET / 10 pm CST), join members of the International Commission on the […]
Read MoreThe Interdisciplinary Scientific Committee (ISC) is establishing a Task Force on Shared Molecular Etiologies Underlying Multiple Rare Diseases aiming to address and document the existing challenges in adapting the basket trial approach used in molecularly targeted oncology clinical trials to drugs targeting shared molecular etiologies underlying multiple rare diseases. The Task Force will identify how […]
Read MoreThe Interdisciplinary Scientific Committee (ISC) is establishing a Task Force on Shared Molecular Etiologies Underlying Multiple Rare Diseases aiming to address and document the existing challenges in adapting the basket trial approach used in molecularly targeted oncology clinical trials to drugs targeting shared molecular etiologies underlying multiple rare diseases. The Task Force will identify how […]
Read MoreThe Diagnostics Scientific Committee (DSC) is establishing a Task Force on the Integration of New Technologies for the Diagnosis of Rare Diseases. This activity proposes to identify the most clinically beneficial combination(s) of metabolomic and genomic tests coupled with artificial intelligence methodologies, which would then be prioritized for development of diagnostic standards. This will ultimately […]
Read MoreThe Diagnostics Scientific Committee (DSC) is establishing a Task Force on the Integration of New Technologies for the Diagnosis of Rare Diseases. This activity proposes to identify the most clinically beneficial combination(s) of metabolomic and genomic tests coupled with artificial intelligence methodologies, which would then be prioritized for development of diagnostic standards. This will ultimately […]
Read MoreWe are pleased to announce that FDA will be holding a virtual public meeting on Friday, March 5, 2021, from 9:00 a.m. to 4:00 p.m. EST to highlight strategies to support rare disease product development. Please visit the public meeting page to register for the event, https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/public-meeting-fda-rare-disease-day-2021-03052021-03052021 To learn more about other activities relevant to rare diseases […]
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