Two gene therapies from Sangamo Therapeutics for hemophilia A and B have been granted special regulatory designations by the U.S. Food and Drug Administration (FDA). Sangamo said in a press release that the FDA has granted orphan drug status to SB-525  gene therapy for hemophilia A, providing the company with…
An experimental form of genome editing called clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a faster and cheaper method of treating people with hemophilia, compared to the current DNA editing method. Because hemophilia is due to DNA mutations, the genome editing could be used to efficiently slice at the DNA,…
Family planning and pregnancy for women with hemophilia Women who are hemophilia carriers (women who have a gene mutation in one of their X chromosomes), may have mild bleeding symptoms due to reduced clotting factor levels in their blood. It is important to determine a woman’s carrier status in order to…
Hemophilia presents challenges to daily life, but improvements in treatment have made it increasingly possible for people with the bleeding disorder to live a normal life. Hemophilia treatment centers Among the most important resources available to patients and their families are specialized hemophilia treatment centers or HTCs. A network…
In recognition of the World Hemophilia Day, BDI Pharma employees company-wide united to raise awareness and money to support those affected by bleeding disorders. Events were held April 17 at the company’s South Carolina, Texas and Kansas sites, with employees joining remotely to raise money to support the WFH (World Federation of Hemophilia) by wearing…
In the U.S., hemophilia A is estimated to affect one in every 5,000 male births, hemophilia B one in 25,000 male births, and hemophilia C one in every 100,000 people (males and females). Hemophilia A is thus four times more common than hemophilia B, and about 10 times more common than…
In this simple animated video from CSL Behring US, hemophilia is explained in easy-to-understand language aimed at children and caregivers of newly diagnosed hemophilia patients. MORE: Explaining hemophilia A and hemophilia B The narrator shares the basic difference between hemophilia A and hemophilia B, and that…
Hemophilia is a rare disorder where a person’s blood lacks a factor which enables it to clot, leading to excessive bleeding that can become life-threatening. This bleeding can occur internally or externally. According to the World Federation of Hemophilia and the National Heart, Lung…
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…
Novo Nordisk expects a decision this month by the U.S. Food and Drug Administration (FDA) on its product N9-GP  (nonacog beta pegol) as a treatment for hemophilia B. Approval of the drug may depend on the conclusions the FDA makes regarding safety concerns raised by its blood products advisory committee,…
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