This is an exciting time in biomedical innovation. New and highly innovative therapies are coming to market that promise significant improvements in patient health. They include a range of novel approaches to treating disease, like gene therapy, immunotherapy, and RNA interference. Many of the most transformative new therapies are designed to target serious, unmet medical needs among often small groups of patients.

Take gene therapy, for example. Gene therapies that are expected over the next few years have relatively small patient populations—ranging from 1,100 to 35,000 patients. A new therapy that’s expected to be approved any day by the FDA will treat spinal muscular atrophy, a fatal disease that afflicts an estimated 500 children each year.

How society pays for these medical breakthroughs is becoming an important part of the broader conversation around the cost of prescription drugs. As that discussion unfolds, it’s important to keep a couple things in mind.Continue reading→

The first approved gene therapy for a neuromuscular disease is now  available for children with spinal muscular atrophy (SMA) thanks to AveXis, a Novartis company.

SMA is caused by a mutated or missing survival motor neuron 1 gene (SMN1) that prevents the body from making enough survival motor neuron protein (SMN), ultimately leading to the loss of motor neurons, muscle weakness, and paralysis seen in SMA.

Newborn screening requirements for SMA in Arizona is the next important step.

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Rapid, accurate cancer test is critical for dogs’ treatment and survivalContinue reading→

When testing for such as lung cancer disease, doctors know that tissue biopsies are necessary and potentially life-saving, though the procedures used to gather tissue can lead to dangerous complications, from bleeding to lung collapse. To lower the need for invasive procedures, researchers at the University of Arizona developed a new blood test that can detect most major cancers and have launched a startup, DesertDx, to bring the invention to doctors and their patients.Continue reading→

Cancer is a protean disease, assuming many forms and disguises. Despite enormous strides in research in past decades, some cancers remain persistently lethal.Continue reading→

Recognized for his commitment and contributions he has made to healthcare and the community through his leadership, Rich Boals, retired President and CEO of Blue Cross and Blue Shield of Arizona, was conferred an honorary Doctorate of Humane Letters degree by Northern Arizona University (NAU) at the college’s spring 2019 commencement ceremony on Friday, May 10, 2019, in the university’s J. Lawrence Walkup Skydome.Continue reading→

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Paradigm Diagnostics, a leading precision medicine company focused on comprehensive cancer testing, announced the addition of Mara Aspinall, Managing Director of BlueStone Venture Partners to the Board of Directors as part of the closing of Paradigm’s $15 million Series B fundraising.Continue reading→

WASHINGTON–(BUSINESS WIRE)–Today, the Biotechnology Innovation Organization (BIO) released a new report, The State of Innovation in Highly Prevalent Chronic Diseases Volume IV: Alzheimer’s Disease Therapeutics, the fourth in a series on the innovation landscape of highly prevalent, chronic diseases. This volume takes an in-depth look at the state of innovation for therapeutics in Alzheimer’s disease, which is expected to affect more than 13.8 million people in the United States by 2050 and cost well over $1 trillion annually.Continue reading→