PTC Therapeutics, Inc. (NASDAQ: PTCT) announced the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that is directly administered to the brain. The gene therapy, which will be marketed in the United States with the brand name KEBILIDI™ (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity. Launch preparations are well underway, with centers of excellence already identified and surgeons trained in the procedure to deliver the gene therapy. AADC deficiency is a highly morbid and life-shortening rare genetic disorder that results in the inability to synthesize dopamine, a neurotransmitter essential for motor function. KEBILIDI is a gene replacement therapy that is directly administered to the putamen of the… 

Implantica AG (publ.), a medtech company at the forefront of introducing advanced technology into the body, including the unique device RefluxStop™ for the treatment of acid reflux, a treatment field with 1 billion sufferers, announces the submission of the second clinical module of the Premarket Approval (PMA) application to the US FDA for RefluxStop™ along with responses to the FDA’s findings from the first module. The second module submitted earlier this week for the RefluxStop™ PMA is the most crucial module of the three-module process for FDA approval. The outcome of our clinical study submitted to FDA shows fantastic positive results, which has echoed around the surgical community, fueling a vibrating interest in RefluxStop among surgeons and GIs all around the world and especially in the U.S. Together with Module 2, responses to the FDA’s findings from Module 1 were… 

Suvoda, a global clinical trial technology company specializing in complex studies in therapeutic areas like oncology, central nervous system (CNS), and rare diseases, announced the receipt of a patent from the US Patent Office for the Suvoda Questionnaire Definition Language (SQDL), part of the software architecture of its eCOA (electronic Clinical Outcome Assessment) product. The patent demonstrates the power of Suvoda eCOA: expedited questionnaire creation, translation, localization, and deployment so clinical trial sponsors can launch their studies more efficiently. While eCOA is traditionally a bottleneck in clinical trial implementation, Suvoda eCOA and its SQDL tool enable sponsors to deliver high quality questionnaires in a matter of hours, instead of days. 

On November 7th 2024, the U.S. Food and Drug Administration (FDA) granted icometrix clearance for icobrain aria, the first and only AI software approved for detecting, measuring and grading amyloid-related imaging abnormalities (ARIA), a potentially harmful side effect of new amyloid-targeting therapies. A large study, needed for FDA clearance, demonstrated that the use of icobrain aria significantly increases the accuracy of ARIA assessments by radiologists and hence allows for safer use of new amyloid-beta targeting therapies for Alzheimer’s disease patients. The first disease-modifying therapies for Alzheimer’s disease have come to the market over the previous years – providing hope to the millions affected by Alzheimer’s disease. These therapies have been linked to the development of specific brain abnormalities known as Amyloid-Related Imaging Abnormalities (ARIA), which, although rare, can lead to neurological complications.Regular brain MRI scans are needed to assess ARIA,…