Topics cited include the high costs of orphan drugs, the importance of a firm diagnosis and adherence to therapy by patients with rare diseases, and the need to create a diagnosis-based registry for rare diseases.EBSCO_AspLancet
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A population-based registry as a source of health indicators for rare diseases: the ten-year experience of the Veneto Region’s rare diseases registry Background Although rare diseases have become a major public health issue, there is a paucity of population-based data on rare diseases. The aim...
PHOENIX — SubcutaneousefgartigimodPH20 SC shows efficacy and tolerability in the treatment ofchronic inflammatory demyelinating polyneuropathy(CIDP), providing a convenient alternative to intravenous and other older treatments for this rare disease. "This was the largest study of CIDP that has been per...
Cameroon received its first batch of China's Sinopharm COVID-19 vaccines on Sunday, the first COVID-19 vaccine shipment for the country since the start of the pandemic. Error loading player: No playable sources found 00:43 Mauritius also received its first batch of China...
We repeated similar analyses for a cohort of 94 trios that underwent whole-genome sequencing (WGS) to ~30X coverage, as part of the Rare Genomes Project (“Methods”). In order to explore additional options for WGS variant filtering, we considered calls from DeepVariant12that were joint-genoty...
CHICAGO — Biosimilars made in India are as safe and effective for retinal vascular diseases as the American-made originator drugs, but cost about 40% less, according to a comparative analysis. "Biosimilars could become the future for biotherapy in retinal diseases," said Alay Banker, MD, from...
Effective Market Exclusivity of New Molecular Entities for Rare and Non-Rare DiseasesKerr, Kirk
Reads passing each set of alignment criteria were then separately assembled using flye (2.6) with the same parameters: “--min-overlap 1000 --asm-coverage 15 --genome-size 0.5 m --iterations 2” [https://github.com/fenderglass/Flye]58, resulting in two sets of contigs. Finally, both...
March 26, 2012 (Washington, DC) — Despite the stigma it seems to have for some patients and their families, lithium is just as effective for treating mania in older patients with bipolar disorder (BD) as the more commonly used valproate, new research suggests. ...