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hemophilia b inhibitors :: Article Creator Gene Therapy For Hemophilia Becomes A Reality Earlier this year we reported on FDA's approval of valoctocogene roxaparvovec (Roctavian) for adults with severe hemophilia A. In this story, we provide an update on what has happened since. Two gene therapies have now been approved for the treatment of hemophilia following the FDA's June approval of valoctocogene roxaparvovec (Roctavian) as the first gene therapy for hemophilia A -- joining etranacogene dezaparvovec (Hemgenix) for hemophilia B, which was approved late last year. But uptake of these new therapies has been relatively slow. Hemophilia A Approval of valoctocogene roxaparvovec for adults with severe hemophilia A was based on data from the global phase III GENEr8-1 study, in which the therapy reduced the average annualized bleeding rate (ABR) by 52% compared with patients' baseline ABR on routine factor VIII prophylaxis. Mean ...

asian blood disorder :: Article Creator Appeal For Asian Donors To Help Boy With Blood Disorder Ihsan Khan, six, pictured with mum Assia, has aplastic anaemia and needs a bone marrow transplant A family is appealing for donors to find a "life-saving" match for their six-year old son. Ihsan Khan, from Leegomery in Telford, suffers from the rare blood disorder aplastic anaemia and needs a bone marrow transplant. His father, Imran, said a shortage of Asian donors makes finding a match more difficult. Potential donors are being invited to attend screening sessions being held at town mosques. Mr Khan says if no match is found for Ihsan, who is currently undergoing treatment at Birmingham Children's Hospital, the consequences would be severe. "When I come back to this house and I see it empty, he's not here...Emotionally it is very hurtful and draining," he said. "We are hoping we can find a match as ...

mixed race genetic disease :: Article Creator Racial/Ethnic Differences In Autoimmune Disease Prevalence In US Claims/EHR Data This analysis uses claims and electronic health records from 2021 to examine racial and ethnic variations in immune-mediated inflammatory diseases in the United States. ABSTRACT Objectives: Few studies have evaluated racial and ethnic differences in several immune-mediated inflammatory diseases (IMIDs) or overlap syndrome (the co-occurrence of ≥ 2 IMIDs). We assessed associations between race and ethnicity and prevalence of IMIDs and overlap syndrome using US claims and electronic health records from 2021. Study Design: Retrospective cohort study of 10.8 million adults. Methods: We identified the 10 most prevalent IMIDs among frequently discussed IMIDs. We estimated associations between the 5 most prevalent IMIDs and overlap syndrome in Hispanic and non-Hispanic Asian and Black adults using non-Hispanic White adults as...

multiple myeloma and thrombocytopenia :: Article Creator Multiple Myeloma Cured After Hepatitis Treatment Reveals That This Cancer Can Be Caused By Viruses The effect of antiviral treatment in hepatitis C virus- and hepatitis B virus-infected multiple myeloma cohorts. (A) Study design and flowchart of hepatitis B virus (HBV)+ (orange) and hepatitis C virus (HCV)+ (blue) cohorts. A total of 1,367 (HBV+) or 1,192 (HCV+) patients from 49 out of the 73 health care organizations (HCO) of the TriNetX network were included in the cohort of patients diagnosed with multiple myeloma (MM) after HBV or HCV infection. Anti-viral treatments (AVT) were tenofovir disoproxil, lamivudine, peginterferon alfa-2a, interferon alfa-2b, tenofovir alafenamide, entecavir. Anti-HCV treatments were elbasvir, grazoprevir, glecaprevir, pibrentasvir, sofosbuvir, velpatasvir, voxilaprevir, or combinations of these drugs. (B) Survival analysis of the HBV+ and HCV+ cohorts. Number ...