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.
Pharmaceutical Technology
OCTOBER 25, 2022
Additionally, Astellas will obtain an exclusive option for licencing two clinical-stage programmes of Taysha, namely TSHA-102 and TSHA-120, for Rett syndrome and GAN, respectively. Astellas will also receive specific rights linked to any possible change of Taysha’s control.
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pharmaphorum
JANUARY 27, 2023
Clinical trials design and patient input The definition of patient centricity, in fact, and its benefits are now – finally – being defined by patients themselves. “We We talk a lot in clinical trials and drug development about benefit,” Dr Mullen said. But who is benefitting?
XTalks
OCTOBER 7, 2020
A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. The Xtalks White Paper, The Intersection of Rare Disease and Advanced Therapies: What it Means for Clinical Development , is available for free download. “They called him ‘The Boy.’
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.
Camargo
JULY 27, 2021
When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.
Drug Discovery World
AUGUST 28, 2023
DDW’s Diana Spencer speaks to Peter Hamley , Chief Scientific Officer of Samsara Therapeutics, to understand the role autophagy plays in neurodegenerative diseases and how boosting this process could increase healthspan. DS: Samsara’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials.
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