News & Analysis on Clinical Trial Services & Contract Research And Development
Rare and orphan diseases - SSNL
Unfortunately, more than 700 rare diseases affect more than 30 million people in the US alone and with many of them being life threatening, it is still a concern that most still do not have treatments.
Enzyme replacement therapy (ERT) made its debut in 1991, heralding a breakthrough in medical science for individuals and families grappling with rare diseases stemming from single gene mutations.
SSNL: Rare and Orphan digest
OSP had a quickfire interview with Rebecca Starkie, senior global patient engagement director, at Advanced Clinical where she explains the challenges faced by rare disease patients - and suggests ways to improve.
SSNL Rare and Orphan Digest
Element Biosciences, Inc., will share news of its sequencing methodology and host a workshop on higher accuracy sequencing for improved variant detection in Washington DC from today.
SSNL Rare and Orphan Digest
A transaction between Citius Pharmaceuticals and TenX Keane which will result in the formation of Citius Oncology, Inc. is due to close in the first half of next year (2024).
Kathryn Starzyk is an epidemiologist and currently vice president of real-world evidence (RWE) at healthcare technology company, OM1.
PicnicHealth announced recently that it has acquired AllStripes, a platform generating evidence about the rare disease industry community.