Top Story

Factor IX gene therapy safe, effective in severe hemophilia B

November 19, 2014

Gene therapy with a novel AAV8 vector safely elevated Factor IX coagulation activity and reduced bleeding episodes in patients with severe hemophilia B, according to study results.

Amit C. Nathwani, MD, ChB, PhD, of the Katharine Dormandy Hemophilia Centre and Thrombosis Unit at Royal Free Hospital in London, and colleagues sought to evaluate the long-term safety and efficacy of gene therapy mediated by a novel self-complementary adeno-associated virus serotype (AAV8) vector.

In the Journals

Sickle cell trait may increase risk for renal disease in blacks

November 13, 2014
Black individuals who carried sickle cell trait demonstrated increased risk for chronic kidney disease and were more likely to experience declines in estimated…
In the JournalsPerspective

Meta-analysis identifies predictors of chronic childhood immune thrombocytopenia

November 10, 2014
Female gender, older age at presentation and the presence of antinuclear antibodies were among the characteristics that predicted chronic immune thrombocytopenia in…
Barbara P. Yawn, MD, MSc, MSPH GuidelinesPerspective

Guideline strongly recommends hydroxyurea, transfusion therapy for sickle cell disease

HemOnc Today, November 10, 2014
An expert panel convened by the NHLBI strongly recommends hydroxyurea and transfusion therapy for most patients with sickle cell disease, according to a summary of the…
CME
New Advances, New Options: Evaluating the Changing Landscape of Hemophilia

New Advances, New Options: Evaluating the Changing Landscape of Hemophilia to Optimize Patient Outcomes

This activity is supported by an educational grant from Biogen Idec.

The bleeding abnormalities associated with hemophilia can have devastating consequences for patients. Recent advances…
More »
CME CNE
Stem Cell Transplant for AAMDS

Treatment Decision-Making through the Spectrum of MDS: Case 3 Stem Cell Transplant for MDS

An educational presentation from Aplastic Anemia & MDS International Foundation.

MDS is a difficult disease to diagnose and treat. Frequently it is challenging for healthcare providers to explain to…
More »
In the Journals

Factor IX gene therapy safe, effective in severe hemophilia B

November 19, 2014
Gene therapy with a novel AAV8 vector safely elevated Factor IX coagulation activity and reduced bleeding episodes in patients with severe hemophilia…
In the Journals

Sickle cell trait may increase risk for renal disease in blacks

November 13, 2014
Black individuals who carried sickle cell trait demonstrated increased risk for chronic kidney disease and were more likely to experience declines in…
In the JournalsPerspective

Meta-analysis identifies predictors of chronic childhood immune thrombocytopenia

November 10, 2014
Female gender, older age at presentation and the presence of antinuclear antibodies were among the characteristics that predicted chronic immune…
Barbara P. Yawn, MD, MSc, MSPH GuidelinesPerspective

Guideline strongly recommends hydroxyurea, transfusion therapy for sickle cell disease

HemOnc Today, November 10, 2014
An expert panel convened by the NHLBI strongly recommends hydroxyurea and transfusion therapy for most patients with sickle cell disease, according…
In the Journals

Weekly prophylaxis with nonacog beta pegol safe, effective for hemophilia B

October 31, 2014
Nonacog beta pegol effectively treated bleeding episodes and was associated with low annualized bleeding rates in patients with hemophilia B…
FDA approvals

FDA approves Obizur for acquired hemophilia A

October 24, 2014
The FDA approved Obizur for the treatment of bleeding disorders in adults with acquired hemophilia A.The development of acquired hemophilia A —…
In the Journals

Efficacy of VTE chemoprophylaxis in otolaryngology patients varied by subgroup

October 24, 2014
The efficacy and safety of venous thromboembolism chemoprophylaxis for individuals undergoing otolaryngologic surgery varied based on Caprini risk…
In the Journals

Recombinant Factor VIII product increased inhibitor development risk

October 24, 2014
One recombinant Factor VIII product was associated with an increased risk for inhibitor formation compared with similar products for the treatment of…
Linda J. Burns, MD Institution Notes

ASH elects four executive committee members

October 18, 2014
ASH elected four new members to its executive committee, the organization’s governing body.Kenneth C. Anderson, MD, will serve a 1-year term as…
Linda J. Burns, MD In the Journals

Hydroxyurea, transfusions urged for sickle cell disease management

October 17, 2014
The use of hydroxyurea and transfusion therapy is “strongly recommended” for patients with sickle cell disease, according to an…
More Headlines »
morganatic-roan