Rare Disease

Research Offers Clues Into T Cell Differentiation in Worsening of Acute Gut GVHD

August 01, 2020


Previous research has established acute graft-versus-host disease (GVHD) in the gastrointestinal tract as the primary cause of both GVHD severity and mortality, and as a result, emphasis is placed on preventing acute gut GVHD while still preserving the graft-versus-leukemia effect of allogeneic hematopoietic stem cell transplantation.

Disease Management Strategies Among Patients With MPNs

July 30, 2020


Patients with myeloproliferative neoplasms (MPNs) have high disease burden, and management of their disease can vary widely to include watchful waiting, phlebotomy, or drug therapy.

Supplemental Process Can Aid in Assessing Value of Rare Disease Treatments

July 25, 2020


A study looking at the ways in which different countries handle appraisal and reimbursement processes for rare disease treatments (RDTs) revealed that implementing supplemental processes can aid in patient and physician decision making.

Ruxolitinib Improves ORR Over Best Available Therapy in Patients With Chronic GVHD

July 24, 2020


A phase 3 study of ruxolitinib (Jakafi) has met its primary end point of superior overall response rate (ORR) compared with the best available therapy in patients with moderate or severe steroid-refractory or steroid dependent chronic graft-versus-host disease (GVHD).

Therapy Provides Readily Available Stem Cell Source to Treat Rare Genetic Diseases

July 22, 2020


Umbilical cord blood provides a readily available source of stem cells to provide better access to hematopoietic stem cell transplantation to safely and effectively treat various noncancerous genetic disorders in children.

Gaps Remain Between European Regions for Hemophilia Care Despite Improvements

July 17, 2020


Hemophilia care principles have been on the rise in Europe over the course of the last decade, however, differing regions and policies on the continent reveal gaps in access to various treatments and specialty services.

App-Based Meditation Can Alleviate Mental Health Difficulties in Patients With MPNs

July 14, 2020


Using mindfulness meditation apps may provide an effective way to self-manage and reduce depression and anxiety in patients with myeloproliferative neoplasm (MPNs).

Review Calls For Clear Guidance to Ensure Patient Centricity in Orphan Drug Trials

July 12, 2020


Researchers called for new guidance to address the underreporting of patient engagement in rare disease clinical trials after finding a dismal amount of patient-reported outcome measures listed in orphan drug labeling claims.

FDA Approves At-Home Therapy to Treat Myelodysplastic Syndromes

July 11, 2020


A new oral therapy approved by the FDA will provide patients with myelodysplastic syndromes, who typically visit a health care facility for intravenous treatment, with an oral at-home option that can be beneficial during the coronavirus pandemic.

Interim Analysis Finds Benefits of Ruxolitinib to Treat PV in Patients Intolerant/Resistant to Hydroxyurea

July 07, 2020


A 52-week interim analysis confirmed previous findings highlighting the benefits of ruxolitinib in patients with polycythemia vera (PV) who are resistant or intolerant to hydroxyurea. The full study findings with a 24-month follow-up are still to come.