The Impact of RADIANT: A Deep Dive into the Study

Death induced by survival gene elimination (DISE) is a cell death mechanism mediated by short RNAs. And according to a recent study, there may be a correlation between toxic DISE, DNA damage, and neuronal cell death in patients with Alzheimer’s disease. Learn more about the study’s findings and potential implications with Dr. Andrew Wilner and Dr. Marcus Peter, Professor of Medicine in the Division of Hematology and Oncology as well as a Professor of Biochemistry and Molecular Genetics at Northwestern University Feinberg School of Medicine in Chicago.

The diagnosis and management of the rare genetic disease thymidine kinase 2 deficiency (TK2d) can come with many challenges. So to help shed light on those challenges and key management strategies, Dr. Charles Turck speaks with Dr. Bruce Cohen about his experiences and approaches to real-world patient cases in TK2d care. Dr. Cohen is the Chair of the NeuroDevelopmental Science Center at Akron Children’s Hospital in Ohio.

Thymidine kinase 2 deficiency (TK2d) is a rare, genetic condition with nonspecific symptoms that wouldn’t lead to a definitive etiological diagnosis, underscoring the important role genetic testing plays in diagnosing patients. Learn more about the common clinical manifestations of TK2d and the diagnostic process with Dr. Charles Turck and Dr. Austin Larson, Assistant Professor in the Section of Genetics and Metabolism within the Department of Pediatrics at the University of Colorado School of Medicine.

Thymidine kinase 2 deficiency is a very rare disorder that presents several diagnostic and treatment challenges. Learn more about those challenges along with the clinical manifestations of thymidine kinase 2 deficiency and the importance of taking a multidisciplinary approach to care with Dr. Bruce H. Cohen, Chair of the NeuroDevelopmental Science Center at Akron Children’s Hospital.

Over the last 15 years, the monogenic diabetes registry now has over 4,000 patients involved, and from that, the RADIANT study was started to identify new kinds of rare and atypical diabetes. So how can genetics testing and the RADIANT study provide atypical diabetes patients with better diagnoses and treatment? Learn more with Dr. John Buse and Dr. Louis Philipson, Professor in the Department of Medicine and Pediatrics at the University of Chicago.

A recent study was designed by the International Multiple Sclerosis Genetics Consortium with the goal in mind to find out whether the severity of multiple sclerosis (MS) could be influenced by genetics. Dive in with Dr. Sergio Baranzini, Distinguished Professor of Neurology at the University of California San Fransico and Co-Senior Author of the study.

Scientists at Temple University’s Lewis Katz School of Medicine have identified a novel gene-editing strategy aimed at eliminating HIV-1 infection with no adverse effects on cell mortality. Find out how this recent discovery could get us one step closer to a cure for HIV with Dr. Charles Turck and Dr. Kamel Khalili, the Laura H. Carnell Professor in the Department of Neuroscience and Chair of the Department of Microbiology, Immunology & Inflammation.

Approximately 165,700 people in the United States are affected by inherited retinal diseases (IRDs) and treatment options for these conditions are limited. So what role does genetic testing play in the patient journey of people living with IRDs? Join Dr. Charles Turck as he dives into this topic with Dr. Mark Pennesi, Professor of Ophthalmology and an IRD specialist affiliated with the Casey Eye Institute at the Oregon Health and Science University School of Medicine.

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This Expert Exchange webcast is designed to help improve clinicians’ ability to apply pharmacologic and non-pharmacologic strategies in the management of patients with ADPKD.

Hereditary angioedema (HAE) is a rare genetic disease that can cause recurrent, unpredictable, and potentially life-threatening attacks of swelling in the body.² The unpredictability of these attacks is a significant burden on patients.³ Fortunately, preventive treatments like TAKHZYRO^® (lanadelumab-flyo) can help reduce the frequency and severity of HAE attacks.¹ Here to share key information and data on this treatment option is Dr. Timothy Craig, Professor of Pulmonary, Allergy, and Critical Care Medicine at Pennsylvania State University.

Indication
TAKHZYRO^® (lanadelumab-flyo) is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients ≥2 years of age.

Important Safety Information
Hypersensitivity reactions have been observed. In case of a severe hypersensitivity reaction, discontinue TAKHZYRO administration and institute appropriate treatment.

Adverse Reactions: The most commonly observed adverse reactions (≥10%) associated with TAKHZYRO were injection site reactions consisting mainly of pain, erythema, and bruising at the injection site; upper respiratory infection; headache; rash; dizziness; diarrhea; and myalgia. Less common adverse reactions observed included elevated levels of transaminases; one patient discontinued the trial for elevated transaminases.

Use in Specific Populations: The safety and efficacy of TAKHZYRO in pediatric patients <2 years of age have not been established.

No data are available on TAKHZYRO …