Shire’s Experimental Gene Therapy Delivery Strategies Show Potential for Hemophilia A and B
Shire is developing a potential new strategy to improve the effectiveness of genetic delivery of clotting factor VIII (FVIII)…
Articles by Alice Melão
New Project Aims to Set Effectiveness Guidelines for Hemophilia Gene Therapy Trials
An international team has joined efforts to establish guidelines for effectiveness and outcome measurements regarding gene therapies in hemophilia. The…
FDA Approves Rebinyn for Hemophilia B Treatment
Novo Nordisk’s recombinant coagulation factor IX nonacog beta pegol (N9-GP), under the U.S. trade name Rebinyn, has been approved…
Initial Results from Phase 3 Hemophilia Trial of Emicizumab in Children Look Promising
First results from the Phase 3 HAVEN 2 study evaluating the effectiveness and safety of emicizumab in children younger than 12…
So Far, Dimension’s DTX101 Clinical Trial Shows Promising Results For Hemophilia B Patients
Dimension Therapeutics’ gene therapy candidate drug DTX101 shows promising results by improving the levels of blood clotting factor IX (FIX)…
Researchers Review Guidelines on Primary Prophylaxis in Hemophiliac Children
Recent studies have addressed the importance of preventive, or prophylatic, treatment in severe cases of hemophilia, and its long-term benefits…
Extended Trial Confirms Alprolix’s Ability to Prevent Bleeding Episodes
A Phase 3 extension trial has confirmed Alprolix’s long-term ability to prevent bleeding episodes in hemophilia B patients, and its safety. Those…
Hemophilia Patients End Up at Emergency Rooms Most Often for Hypertension and Injuries
Adult hemophilia patients use hospital emergency departments mainly due to cardiovascular events, while pediatric patients most frequently complain of injuries, according…
Potential Hemophilia B Therapy Shows Promising Results in Mouse Study
A new delivery mechanism of mRNA therapy was found to efficiently reduce hemophilia B symptoms by correcting the protein deficiency that…
Standardized Outcome Measures in Clinical Practice May Improve Hemophiliac Patient Care
A recent survey has shown that the use of appropriate standardized outcome measures of hemophiliac patient treatments in routine clinical practice…