Q: Severe combined immunodeficiency (SCID) is characterized by severely impaired T-cell development and is fatal without treatment. What is the current treatment of infants identified as having SCID, and what is their likely clinical outcome if treated shortly after birth? A: • Overall, with improvements in...
Q: Genomics, Digital Health, Big Data, and Artificial Intelligence are some of the newest technologies/fields that are reshaping medicine and healthcare. How will these technologies impact healthcare and in particular individualized medicine? Are we there yet in terms of tangible impact, or is a good...
The field of precision medicine is moving forward in full speed. We are all witnessing it! Whether it is with the launch of various precision medicine/population sequencing studies, Or immunotherapy getting to be the new standard of care, and with it the potential for personally tailored treatments...
Q: What does your role entail as the director of the MolDX program at Palmetto GBA? A: The job directing MolDX is multifaceted; first and foremost the MolDX program is responsible for assessing molecular diagnostic tests on the market and makes coverage and pricing determinations for...
Q: The CBER’s Regenerative Medicine Advanced Therapy Designation program has been very successful, with about 100 requests for designation in the two years of its existence. Can you please tell us about the program and how it was put together? A: The Regenerative Medicine Advanced Therapy...
Q: What patient data do we need to better understand the underlying cause of disease and how to prevent it? A: Medicine at present is highly underdetermined and data poor. To be precise, one must be comprehensive, so medicine (with our consent) will use not only...
A big ‘Thank You’ to all of our presenters and attendees for celebrating 10 years of precision medicine progress with us! PMWC 2019 Silicon Valley was attended by 2000 participants from 35 countries, which included over 400 speakers in 5 parallel tracks!...
Q: Tell us more about your organization/company. What patient population are you serving and which services are you specializing in? A: Ambry Genetics is a recognized leader in high quality complex genetic testing. We seek to find the genomic cause or contributors to rare diseases,...
Q: What is the state of big data and analytics in healthcare, and how to best use the reams of data available? A: More than ever, Healthcare organizations are achieving measurable value through use of their data and analytics assets. There is more raw material...
Q: There are various new, emerging technologies that bring us closer towards a cure for life-threatening disorders such as cancer, HIV, or Huntington’s disease. Prominent examples include the popular gene editing tool CRISPR or new and improved cell and gene therapies. By when can we...
