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Biology opens up new frontiers in medicine to cure genetic diseases: Kiran Mazumdar Shaw

AuroBlog - Aurous Healthcare Clinical Trials blog

We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. In […]

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Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

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.

Genetics 189
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Abu Dhabi DoH signs new deal to accelerate development in life sciences

Pharmaceutical Technology

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.

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Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

XTalks

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

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.

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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

While the table lists acceptable surrogate endpoints for trials of more common conditions, such as asthma, type 2 diabetes and various cancer types, the document also provides key information to developers of certain rare disease therapies. Individuals with this disease are deficient in ?-galactosidase Mutations in the gene encoding ?-Gal

Trials 98
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Patient centricity and the changing pharmaceutical vista

pharmaphorum

In the morning, I can be talking about gene therapy for hearing loss, for example, [and] in the afternoon, I can be talking about the treatment of a very specific tumour. million people in the UK that have a rare disease,” she continued, “and that’s where a lot of the innovation is, in more of these rare genetic diseases.