Rare Disease

CLAD, Pulmonary GVHD Have Similar Clinical Traits, Research Needs

May 19th 2022, 7:14pm


Fine-tuning clinical phenotypes and identifying effective treatments remain challenging in chronic lung allograft dysfunction (CLAD) after lung transplantation and chronic graft-versus-host disease (GVHD) after allogeneic hematopoietic stem-cell transplantation.

RAS Inhibitors May Hold Potential as Preventive for Acute Chest Syndrome in Sickle Cell Disease

May 15th 2022, 1:21pm


A retrospective study assessed hospital readmission rates in patients with sickle cell disease and acute chest syndrome who took angiotensin-converting enzyme inhibitors or angiotensin II receptor blockers vs those who did not.

New FDA Program to Focus on Driving Rare Disease Drug Development

May 12th 2022, 3:54pm


In an effort to bolster development of treatments for rare diseases, the FDA announced the creation of the new Accelerating Rare disease Cures (ARC) program.

Review Highlights Treatment Landscape, Insights on Pregnancy Management for ET or PV

May 8th 2022, 8:59pm


Polycythemia vera and essential thrombocythemia are both slow-progressing conditions and treated mainly with symptom management, but novel agents and gene targets have changed the landscape in recent years.

Exploring Payer Coverage Decisions Following FDA Novel Drug Approvals

May 3rd 2022, 8:31pm


On this episode of Managed Care Cast, Ari D. Panzer, BS, lead author and researcher, then at Tufts Medical Center—now at Duke University—discusses the findings from his team’s investigation into coverage decisions by health plan insurers of the 66 drugs approved by the FDA in 2018.

Poorer Outcomes Associated With Recurrent aGVHD Warrant Independent Recognition, Says Study

May 2nd 2022, 1:15pm


After a median follow-up of 535 days, analysis showed that recurrent acute graft-versus-host disease (RaGVHD) carried several implications for survival, leading the researchers to call for further risk stratification of aGVHD.

Real-world Study Shows Ruxolitinib May Reduce Arterial Thrombosis in Polycythemia Vera

April 27th 2022, 11:12am


Previous studies have shown ruxolitinib effective in symptom relief and hematocrit control for hydroxyurea-resistant polycythemia vera, but its role in disease progression is not yet clear.

MoCA Scores in HD Correlate With Caudate Atrophy, Cortical Thinning, Says New Research

April 24th 2022, 5:53pm


The findings suggest that the tool can be used to not only assess cognitive impairment in Huntington disease (HD) but also to detect brain atrophy patterns associated with cognitive status in these patients.

Decreased Levels of Zinc Associated With Worse SCD Severity in Children, Adolescents

April 22nd 2022, 9:32pm


A review of 41 articles indicated that leveraging zinc supplementation in patients with sickle cell disease (SCD) is a safe and potentially effective approach for these patients.

Study Demonstrates Potential of Composite End Point in Assessing Treatment for PNH

April 19th 2022, 1:25pm


A recent study found that a composite end point developed by the researchers gave a single and simultaneous measurement of the overall benefit of treatment for paroxysmal nocturnal hemoglobinuria (PNH).