WFH Gene Therapy Registry: now live!
The World Federation of Hemophilia (WFH) has developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR). The launch of the WFH GTR coincides with the first approvals of gene therapy for hemophilia by both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
From the CDC: New Hemophilia Key Findings Now Available
On December 1, the CDC recently released a new Key Findings summary of a recent scientific publication of women and girls who have hemophilia. The CDC reports, "Hemophilia primarily affects men, but women can have hemophilia, too. It was once thought that only men could have hemophilia and women could only pass on the gene that causes hemophilia […]
Children’s National Hospital Rare Disease Institute and Takeda Partner to Standardize Care for Patients With Rare Diseases
The following is an excerpt from a press release from Takeda. Read the press release in its entirety here. Children’s National Hospital and Takeda Pharmaceutical Company Limited announce the creation of the Rare Disease Clinical Activity Protocols (Rare-CAP) program, which will establish a networked system for the development, dissemination and curation of protocols to help […]
BD Announces Voluntary Recall of ChloraPrep™ 3 mL Applicator in Specific U.S. Territories and Countries
NOTE:Â The recall does not apply to any states in the United States, it only applies to the U.S. territories of Puerto Rico, Guam, U.S. Virgin Islands, Northern Mariana Islands and American Samoa. Click Below to Translate Page to Spanish. [prisna-google-website-translator] BD (Becton, Dickinson and Company), a global medical technology company, announced a voluntary recall on […]
WFH & NHF Issue Joint Statement Regarding Two Deaths Reported in Orphan Disease Gene Therapy Clinical Trial
The following is information from the World Federation of Hemophilia. A joint World Federation of Hemophilia (WFH) and National Hemophilia Foundation (NHF) statement* is issued on two deaths reported in orphan disease gene therapy clinical trial (*on behalf of the WFH Coagulation Product Safety, Supply, and Access Committee):We have become aware of two patient deaths […]
FDA Communication: Important Information for Potential Donors of Blood and Blood Products
The following is an excerpt from a recent communication from the United States Food and Drug Administration. Read the full communication here:The Food and Drug Administration (FDA) would like to remind the public that individuals who have ever tested positive for HIV (the virus that causes AIDS) should not donate blood, because of the potential […]
FDA Identifies Biomarker for Immune Response to FVIII Products Used to Treat Hemophilia A
The following is a press release from the FDA. Read the press release on the FDA website here. Scientists at the U.S. Food and Drug Administration developed a technique that enables them to determine whether immune system cells called dendritic cells appear primed to trigger the production of antibodies against Factor VIII (FVIII) products used to […]
FDA Issues a MedWatch Safety Alert Regarding Certain Hepatitis C Medications
The following is an excerpt from a FDA MedWatch Safety Alert. Read the full safety announcement here.FDA has received reports that the use of Mavyret, Zepatier, or Vosevi to treat chronic Hepatitis C in patients with moderate to severe liver impairment has resulted in rare cases of worsening liver function or liver failure.FDA identified 63 […]
FDA, CDC identify potential infection risk prompting voluntary recall of syringes by BD Medical
The following is an alert from BD Medical. Read the full consumer letter with detailed product information here.  Out of an abundance of caution and in the interest of public health, BD is voluntarily recalling certain lots of BD PosiFlush™ Heparin Lock Flush and BD™ Pre-Filled Normal Saline Flush syringes due to a potential for […]
MASAC Issues Safety Information Update on Emicizumab (HEMLIBRA)
The National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) has released a safety information update about Emicizumab (Hemlibra), a new subcutaneous treatment for hemophilia A patients with inhibitors. This update has been released following the first report of an anti-drug antibody to Emicizumab. MASAC’s update issues recommendations for providers and patients in case of […]