Rare Disease

Several Treatment Classes Show Promise in Treatment of PV

April 15th 2021, 6:30pm

Article

The review comes amid growing attention directed at identifying treatments that can prevent progression of polycythemia vera (PV) to myelofibrosis or an aggressive form of acute myeloid leukemia.

What We're Reading: UK Variant Not Linked With Severe Disease; COVID-19 Hospitalization in Children; Therapy Setbacks in Huntington Disease

April 13th 2021, 2:30pm

Article

A new study finds no link between the UK COVID-19 variant and more severe disease; study finds more than 1 in 10 US children with COVID-19 hospitalized; therapy setbacks in Huntington disease.

Ruxolitinib Drives High Rate of Transplantation for Patients With Myelofibrosis

April 9th 2021, 10:15am

Article

The study also indicated that the type of transplantation donor impacted outcomes following the procedure.

Case of Shulman Syndrome Highlights Importance of MRI in cGVHD Treatment Decisions

April 3rd 2021, 5:30pm

Article

According to authors, magnetic resonance imaging (MRI) should be used when incomplete signs of the rare complication are presented to help guide the skin biopsy.

Research Highlights Mast Cell Involvement in cGVHD

April 2nd 2021, 6:00pm

Article

According to a recent review, data suggest mast cells have an important role in tissue homeostasis and wound healing, with mass cell dysregulation potentially leading to fibrotic disease.

Report Lauds Orphan Drug Act but Notes Most Therapies Are Blocked From Competition

March 28th 2021, 12:15am

Article

Although the FDA has approved 599 orphan drugs since 1983, patent laws and other exclusivities keep most from any competition.

Genome Sequencing Producing Precise Diagnoses for Range of Rare Diseases

March 26th 2021, 1:30pm

Article

A Swedish study analyzed 5 years’ worth of data from over 3000 patients, 40% of whom received a molecular diagnosis.

Assessing Novel Treatments to Reduce Thrombosis in PV, ET Requires New Approaches

March 20th 2021, 12:30pm

Article

According to the researchers, few treatments have been able to reduce the thrombotic burden in patients with polycythemia vera (PV) and essential thrombocytopenia (ET), and progress on this front has been hindered by a lack of studies designed to assess a treatment’s impact on thrombotic events.

Gene Expression Profiles May Provide Complementary Prognostic Information for MF

March 19th 2021, 3:30pm

Article

The researchers of the study say their findings may aid the hematological community in determining the value of integrating gene expression profiles in contemporary prognostic models to better identify high-risk patients with a poor prognosis.

How Does Race Play a Role in Complications, Survival in MPN?

March 13th 2021, 8:00pm

Article

While the findings of the small study indicate that there was no statistically significant difference in the rate of vascular complications between White and non-White patients with disease, there were differences in survival.