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Jan 14, 2025 · Be Bio raises $92M to advance hemophilia B treatment BE-101. Be Biopharma has raised $92 million in series C financing to support the first-in-human Phase 1/2 trial of BE-101, the company’s B-cell therapy candidate for hemophilia B. Proceeds from the funding round will be partly used to generate proof-of concept data from the BeCoMe-9 trial (NCT06611436), which…
6 famous people with hemophilia – Hemophilia News Today
Aug 24, 2017 · Queen Victoria. Queen Victoria was a carrier of hemophilia B, a disease that she passed on to two of her daughters and one of her sons, resulting in high-profile royal cases of hemophilia in Germany, Russia, and Spain.Her son, Prince Leopold, died in 1884 following a fall. Through her daughter’s marriages into other European royal families, more monarchs were born with the disease, including ...
Hemophilia A, B prophylaxis therapy Hympavzi may soon be …
Oct 25, 2024 · Developer Pfizer has tagged Hympavzi with a wholesale acquisition cost of $795,600 per year, which compares to the “pricing for prophylactic treatments for hemophilia A or B now on the market,” Younos Abdulsattar, Pfizer’s U.S. rare disease medical lead, said in a Q&A with Hemophilia News Today.. Patients’ actual out-of-pocket costs will vary based on their insurance coverage and many ...
Liver cancer seen as common risk for men with hemophilia and HIV
Dec 3, 2024 · Clotting factor deficiency places patients at an increased risk of excessive bleeding, a characteristic disease symptom that is generally managed and prevented with hemophilia treatments like replacement therapies, which provide a version of the clotting protein patients lack.. Estimates indicate that around 30% of hemophilia patients in Japan contracted HIV, the virus that causes AIDS ...
FDA is reviewing fitusiran application for hemophilia A, B, with...
Jun 26, 2024 · The U.S. Food and Drug Administration (FDA) is reviewing a request to approve fitusiran, an under-the-skin injection therapy, as a treatment for adults and adolescents with hemophilia A or hemophilia B, with or without inhibitors.. A decision is expected by March 28, 2025, according to fitusiran’s developer Sanofi. “We look forward to working in partnership with regulatory agencies to keep ...
Centessa discontinues development of SerpinPC for hemophilia
Nov 15, 2024 · The company was also conducting a long-term open-label extension study called PRESent-6 (NCT06568302) to evaluate the safety and efficacy of SerpinPC for up to 25 months in patients who had completed other trials of the therapy.. SerpinPC was granted fast track and orphan drug status by the U.S. Food and Drug Administration for hemophilia B. The designations are intended to speed the ...
Hemlibra safely prevented bleeds in acquired hemophilia A...
Oct 1, 2024 · The treatment also led to shorter hospital stays, helped patients maintain their daily living activities, and contributed to lower the doses of bypassing agents, reducing healthcare costs.Additional “real-world data from more patients and long-term follow-up are needed to confirm these findings,” the researchers said.
Transformed by Mounjaro: An update on my weight loss journey
Mar 22, 2024 · In a previous column, I shared my journey of embarking on a weight loss program using the type 2 diabetes medication Mounjaro.I’ve used it off-label, as I do not have diabetes. Last November, the U.S. Food and Drug Administration approved the same medication, tirzepatide, for weight loss under the brand name Zepbound.. Inspired by Oprah Winfrey’s recent TV special “An Oprah Special ...
Hemophilia prognosis and life expectancy
Hemophilia is classified as mild, moderate, or severe, depending on the level of clotting factor in the blood. About 70% of those with hemophilia have the severe form. The most important life-threatening complications of the disease are bleeding in the skull and hemorrhages in soft tissue around airways or other internal organs.
Replacement Therapy - Hemophilia News Today
Replacement therapy is one of the standard therapies used to treat hemophilia, a genetic disorder caused by the absence or defects in blood clotting factors.Hemophilia makes patients highly susceptible to bleeding due to a compromised cascade of clotting reactions that are necessary to “seal” injuries.. Replacement therapy involves supplying the missing clotting factor to …