Regulatory agencies in the U.S. and Europe are reviewing applications seeking the approval of fidanacogene elaparvovec, an experimental one-time gene therapy being developed by Pfizer for the treatment of hemophilia B in adults. The U.S. Food and Drug Administration (FDA) expects to complete the review process and…
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TRM-201 (rofecoxib) is an oral nonsteroidal anti-inflammatory drug (NSAID) being developed by Tremeau Pharmaceuticals to treat hemophilic arthropathy, a degenerative joint disease that can occur due to recurrent joint bleeds in people with hemophilia.
2seventy Bio has expanded its collaboration with Novo Nordisk to develop a gene-editing approach to treat hemophilia A. Under this agreement, Novo Nordisk has the option to exclusively license 2seventy Bio’s proprietary mRNA-based megaTAL technology — customizable enzymes designed to recognize and cut specific DNA regions.
A single administration of AMT-061, an investigational gene therapy for people with severe and moderately severe hemophilia B, increased therapeutic levels of factor IX (FIX) in all patients enrolled in uniQure’s ongoing Phase 2b trial, the company announced. Ultimately, this trial’s purpose is to confirm the dose of AMT-061…
This morning I walked with my youngest son, Caeleb, into his school. He depended on a walker to help relieve the pain he felt in his right ankle and knee. The ravages of multiple internal bleeding episodes have left my son with tremendous pain. His ability to walk…
Older patients with hemophilia have lower rates of polypharmacy, or use of multiple medications, and of drug-drug interactions than people of the same age without the disease, a review study reports. This is likely due to their regular risk assessments at hemophilia treatment centers (HTCs) and coordination of care…
Hemophilia joint health care involves treatments, physiotherapy, and lifestyle adjustments such as doing low-impact exercises, following a well-balanced nutrition plan, and maintaining good mental health.
Novo Nordisk A/S has launched HemaGo XChange, a new web-based service portal for people with bleeding disorders. This platform will address the overwhelming demands that patients with hemophilia might face in the monitoring and sharing of information about their disease. HemaGo XChange allows patients to monitor and share treatment…
I recently picked up a fascinating book, “The Courage to Be Disliked,” by Ichiro Kishimi and Fumitake Koga. The book dives into the philosophy of Alfred Adler, a pioneering figure in individual psychology. Adler’s ideas stand in contrast to the older, trauma-focused theories of…