Rare Disease

Novel Ruxolitinib Combo Shows Promise in Myelofibrosis

June 18th 2021, 1:45pm


Presented as an abstract at the European Hematology Association 2021 Virtual Congress, the study results showed the novel combination resulted in early disease modification among 34 patients.

Greater Understanding of PV, ET Drives Potential New Treatment Options

June 12th 2021, 3:15pm


The novel agents are a result of a greater understanding of the conditions and may modify the diseases’ course and cytoreduction, researchers said.

European Data Initiative Aims to Solve Rare Diseases

June 10th 2021, 6:30pm


Using the new data initiative, researchers hope to diagnose unsolved cases of rare diseases.

Peer-Delivered Intervention Can Increase Patients’ Acceptance of Rare Diseases

June 5th 2021, 5:30pm


Results of a clinical trial indicate peer-delivered interventions can help patients with rare diseases achieve disease acceptance.

Early Initiation of Ruxolitinib Reduces Risk, Severity of aGVHD

June 3rd 2021, 7:03pm


The study of 57 patients with acute graft-versus-host disease (aGVHD) also showed that patients tolerated the treatment, offering promise for preventing and managing the complication of transplantation, which affects up to half of patients.

Novel Approaches Needed to Mitigate Myelofibrosis Persistence, Say Researchers

May 31st 2021, 11:15am


According to researchers, type 2 inhibitors do not seem to result in persistence of disease. In one mouse model, the inhibitor type was shown to also have a greater effect on disease biology.

Caring for Children With AADC-d Impacts Parental Ability to Work, Study Shows

May 28th 2021, 3:45pm


The study portrays the intense need for daily care experienced by children with aromatic L-Amino acid decarboxylase deficiency (AADC-d) and their families.

Trial Finds Rituximab Is Superior to Mycophenolate Mofetil in Treating Pemphigus Vulgaris

May 26th 2021, 7:30pm


A phase 3 trial explored which treatment option for pemphigus vulgaris, a rare skin disease, resulted in better outcomes after 52 weeks.

With No Treatment for Tangier Disease, Management Focuses on Preventing Atherosclerosis

May 22nd 2021, 1:00pm


There are no curative treatments for Tangier disease, but gene therapy for ABCA1 has been highlighted as a potential avenue for treating the disease, say the researchers of the paper.

The Economic Burden and Impact on HRQOL of Sickle Cell Disease

May 21st 2021, 6:52pm


Patients with sickle cell disease face substantial health care utilization and impaired health-related quality of life (HRQOL).