Focus on Rare Diseases (October 2016, Volume 2, Issue 10.1)

October 2016 Special Edition

Volume 2, Issue 10.1

This supplement features FDA updates, clinical trials, policy updates, and the latest research in hematologic rare diseases.

Table of Contents

Focus on Rare Diseases

Policy Updates

Saturday, October 1st, 2016
ASH Calls for Payment Innovation in Rare Diseases Such as Sickle Cell Disease The Center for Medicare and Medicaid Innovation (CMMI), a branch of the...

Focus on Rare Diseases

FDA Update: Recent Approvals and Designations for Orphan Drugs

Saturday, October 1st, 2016
In instances where an investigational drug would be the first available treatment or would have advantages over existing treatments, the U.S. Food and Drug...

Focus on Rare Diseases

Clinical Trials for Rare Diseases

Saturday, October 1st, 2016
Below, we take a look at ongoing clinical trials for rare diseases. In addition to searching for new therapeutic targets and developing novel drugs...

Focus on Rare Diseases

Real-World Study Suggests Acquired Hemophilia A Patients Can Receive Lower Doses of Recombinant Porcine...

Saturday, October 1st, 2016
A retrospective study presented at the Thrombosis and Hemostasis Societies of North America 2016 Summit provided real-world data on recombinant porcine factor VIII (rpFVIII)...

Bortezomib Leads to More Complete Remissions in Patients With High-Risk Follicular Lymphoma

Saturday, October 1st, 2016
With few monotherapy treatment options available for patients with high-risk follicular lymphoma (FL), investigators are evaluating combinations with agents approved for other indications to...

A Look at Patient Outcomes in Overlapping Multiple Myeloma and Chronic Lymphocytic Leukemia

Saturday, October 1st, 2016
Though multiple myeloma (MM) and chronic lymphocytic leukemia (CLL) have overlapping epidemiologic features and originate from mature B cells, the simultaneous occurrence of both...

Masitinib Improves Symptom Burden, Responses in Patients with Severe Systemic Mastocytosis

Saturday, October 1st, 2016
Masitinib, a selective oral tyrosine kinase inhibitor (TKI) targeting wild-type KIT, LYN, and FYN, reduced the severity of a diverse range of symptoms in...

Long-Term Safety and Efficacy Data for Pacritinib in Myelofibrosis: Updates from PERSIST-1

Saturday, October 1st, 2016
In two updated analyses from the phase III PERSIST-1 study presented at the 2016 ASCO Annual Meeting, authors presented new data about the safety...

Examining Mogamulizumab for T-Cell Leukemia and Lymphoma

Saturday, October 1st, 2016
Patients with adult T-cell leukemia and lymphoma (ATLL), a rare T-cell malignancy caused by the human T lymphotropic virus type 1 (HTLV-1), have limited...

SL-401 Improves Overall Response Rate for Blastic Plasmacytoid Dendritic Cell Neoplasm

Saturday, October 1st, 2016
SL-401, a targeted therapy directed to the interleukin-3 receptor CD123, was shown to improve overall response (ORR) and complete response (CR) rates in patients...

Clotting Factor Deficiencies Associated With Excess Bleeding Risk, Regardless of Factor Levels

Saturday, October 1st, 2016
Women who have or are carriers for factor VIII (FVIII) or factor IX (FIX) deficiency often report excessive bleeding, though it is unclear how...

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