AADC Deficiency Study – Targeting the Putamen with Gene Therapy

Dr. Matthew Klein, MD, Chief Development Officer at PTC Therapeutics discusses Aromatic l-amino acid decarboxylase (AADC) deficiency, a very rare genetic disorder, and the results of a study published in “EMBO Molecular Medicine” from experts at leading children’s hospitals around the world, including Boston Children’s Hospital, that describes multiple clinical trials in which a novel gene therapy, AAV2-hAADC, was infused into the putamen of 20 children with AADC deficiency via brain surgery. Following the therapy all showed significant improvements. He also talks about PTC’s rapidly advancing gene therapy pipeline, which includes AAV2-hAADC. 

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PixCell Medical – HemoScreen and Point-of-Care Diagnostics

Hanan Ben-Asher, COO and Business Development at PixCell Medical discusses the development of a novel miniaturized portable hematology analyzer called HemoScreen™, which provides diagnostic results in minutes instead of days. With just one drop of blood and within six minutes, HemoScreen delivers accurate readings of 20 standard blood count parameters, saving patients, clinicians and health systems time and money.​ He also talks about the benefits of using AI in diagnostics, how diagnostic technologies played a major role in battling COVID-19, and how point of care testing is the future of disease maintenance. 

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SPR Therapeutics – SPRINT PNS – Percutaneous Peripheral Nerve Stimulation

Dr. Christopher Gilmore, MD, of the Carolinas Pain Institute and lead author on the multi-center trial of the largest base of patients thus far evaluating SPR Therapeutics‘​ ​​percutaneous ​peripheral ​nerve ​stimulation​ called SPRINT ® PNS for low back pain​ discusses this drug free, minimally invasive ​option​.​ ​​ The treatment is up to 60 days but offers long-term pain relief for many patients dealing with back pain, pain in the extremities, and for chronic and acute applications. 

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American Association of Critical-Care Nurses (AACN)

Beth Wathen, American Association of Critical-Care Nurses (AACN) President and frontline nurse at Children’s Hospital Colorado discusses AACN and what they are doing to make sure nurses’ voices are heard. She talks about a recent survey on the Delta variant’s impact on frontline nurses, and with ERs and ICUs flooded with COVID patients, how it impacts the rest of us. She shares the truth about life and death of the unvaccinated in ICUs and how both the unvaccinated and vaccinated people can help support nurses.

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New Methods in Prostate Cancer Detection

Dr. Earle Burgess an oncologist from Charlotte, NC discusses prostate cancer, the second most common form of cancer in American men today. He talks about ways of detection with the prostate-specific antigen (PSA) measured in a blood test and another important yet underrecognized biomarker called prostate-specific membrane antigen (PSMA) that is measured through a PET scan to see where the cancer has spread in the body and gives the patient and the doctor more information on the patients metastatic prostate cancer.

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Zimmer Biomet’s – ROSA Hip System for Robotically-Assisted Direct Anterior Total Hip Surgery

Dr. Atul Kamath, MD, Director, Center for Hip Preservation at the Cleveland Clinic and a ROSA® Hip developer surgeon discusses the recent FDA clearance of the ROSA® Hip System. This marks the fourth robotic system introduced by Zimmer Biomet. It aims to robotically assist surgeons through critical steps of the procedure and allows them to use real-time data and visualization tools with the goal of reducing intra-operative variability and inconsistency. ROSA® Hip is also the latest addition to ZBEdge™, Zimmer Biomet’s suite of digital and robotic technologies designed to seamlessly share data and deliver clinical insights throughout the entire surgical experience with the goal of improving patient outcomes. 

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SpringWorks Therapeutics – Desmoid Tumor Awareness

September is Desmoid Tumor Awareness Month, and given that nearly 30% to 40% of desmoid tumors are initially misdiagnosed, partly due to a lack of awareness, it is important to raise awareness among both patient and doctor communities to better support those who may be living with these devastating tumors. Dr. James (Jim) Cassidy, MD, PhD, Chief Medical Officer at SpringWorks Therapeutics discusses desmoid tumors, also called aggressive fibromatosis or desmoid-type fibromatosis, which are rare and often debilitating and disfiguring soft-tissue tumors with no FDA-approved treatment. He also talks about how SpringWorks is working to advance a potential new treatment option for people with desmoid tumors. 

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Happify Health – Ensemble – Prescription Digital Therapy

One in five people suffer from some form of depression or anxiety, which has been exacerbated by the pandemic. Barriers and limited access to care, coupled with a shortage of mental health physicians has helped spur tremendous growth (and record investments) in digital therapeutics, which is not only filling the gap, but increasingly proving to be a viable option. Chris Wasden, Head of Digital Therapeutics at Happify Health discusses ​these trends and the launch of ​Ensemble, a first-of-its kind prescription digital therapy solution that leverages a myriad of targeted AI, gamification and 100+ Cognitive-Behavioral Therapy (CBT) and mindfulness interventions that was brought to market under the FDA’s Covid Emergency Authorization.​ 

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