pharmaphorum
JANUARY 18, 2023
By designing research with participants involved from the start, and with autonomy as a core principle, we can escape from the often binary and paternalistic views that dominate. And if participants have opted in to be made aware of their risks of genetic diseases, then communication has to be carefully considered.
Worldwide Clinical Trials
JANUARY 24, 2024
Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. In my capstone, I focused on how, if, and when genetic counselors discuss clinical trials with their patients in the context of a counseling session.
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Pharmaceutical Technology
JUNE 16, 2023
The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.
Medical Xpress
JANUARY 20, 2023
The results are published in The Lancet Neurology.
XTalks
MAY 17, 2023
Related: Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall “The BGTC offers promise for rare disease patients around the world,” said Julie Gerberding, MD, CEO of the FNIH, in the press release from the agency.
XTalks
APRIL 24, 2023
Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.
The Pharma Data
MARCH 16, 2021
“Von Hippel-Lindau disease is a rare genetic condition for which there is no systemic treatment option available and is associated with a high risk of cancer development in multiple organs. Research into VHL biology that led to the discovery of HIF-2? About Von Hippel-Lindau Disease and Renal Cell Carcinoma.
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