January 23rd 2024
Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
December 26th 2023
MoCA Scores in HD Correlate With Caudate Atrophy, Cortical Thinning, Says New Research
April 24th 2022The 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.
Read More
Study Demonstrates Potential of Composite End Point in Assessing Treatment for PNH
April 19th 2022A 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).
Read More
ICER Examines Possible Policies Aimed at Sustaining Future Rare Disease Drug Development
April 14th 2022The Institute for Clinical and Economic Review (ICER) and researchers at NORC at the University of Chicago released a white paper that looks at the future of affordability and sustainability of drug development for rare disease through the lens of 4 policy proposals.
Read More
Dr Bernice Kwong Discusses Impact of Delayed Diagnosis, Treatment for GVHD
April 8th 2022Bernice Kwong, MD, clinical professor of dermatology, Stanford University, talks about how later diagnosis and treatment for graft-versus-host disease (GVHD) affects disease progression and overall patient outcomes.
Watch
Case Report and Review Highlight Importance of Identifying Osteolytic Lesions in MPNs
April 1st 2022A recent report details the first reported case of an osteolytic lesion in polycythemia vera and reviews current literature on osteolytic lesions in myeloproliferative neoplasms (MPNs) overall.
Read More
What We’re Reading: Epilepsy Drug Approval; Free COVID-19 Testing Ends; EPA Sued
March 28th 2022The FDA approved a drug to treat a rare form of childhood epilepsy; uninsured Americans will no longer have access to free COVID-19 tests; a conservation group is suing the Environmental Protection Agency (EPA) over failure to protect rivers from pollution.
Read More
What We’re Reading: FDA Reviews ALS Drug; Vaccines Lower Long COVID-19; New COTA President, CEO
March 25th 2022The FDA accepts an application for a drug to treat amyotriphic lateral sclerosis (ALS) for review; vaccines have been found to reduce people’s risk for developing long COVID-19; COTA announces Miruna Sasu, PhD, as its new president and CEO.
Read More
Potential Link Found Between Thrombotic Events, Mortality in Patients With PV, ET
March 13th 2022Patients with polycythemia vera (PV) or essential thrombocythemia (ET) had a higher risk of thrombotic events than the general population, which was associated with mortality in a recent study.
Read More
Pfizer will begin phase 2 and 3 trials of its COVID-19 antiviral pill in children 6 years and older; lawmakers respond after e-cigarette companies began using unregulated synthetic nicotine to get around FDA regulation; the American College of Rheumatology released a new guideline on the management of Kawasaki disease.
Read More
PD-1/PD-L1 Pathway May Hold Promise in MDS/AML, Questions Remain
March 7th 2022A new review article outlines the latest research and results into programmed cell death-1/programmed cell death ligand-1 (PD-1/PD-L1) inhibitors for the treatment of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML).
Read More
Report: Economic Burden of Rare Diseases Is 10 Times Higher Than Mass Market Diseases
March 2nd 2022The economic burden of rare diseases based on direct, indirect, and mortality-related costs is 10 times greater than the burden for mass market diseases, such as diabetes and cardiovascular disease, and this burden increases when no treatment is available.
Read More
Bone Marrow Fibrosis Holds Potential as Independent Prognostic Factor in MDS
February 19th 2022Myelodysplastic syndrome has a variety of clinical presentations, including bone marrow fibrosis. Previously, the presence of fibrosis has not been considered in disease risk scoring in MDS, but recent research suggests it may be a valuable risk factor.
Read More
Ponatinib, High-Dose Chemo Combo Likely to Benefit High-risk BP-CML
January 1st 2022Survival rates for patients with blast-phase chronic myeloid leukemia (BP-CML) are notoriously poor, necessitating effective treatments to fill the care gap for this group who often survive less than 1 year.
Read More
Top 5 Most-Read Rare Disease Articles of 2021
December 20th 2021The top 5 most-read rare disease articles of 2021 on AJMC.com highlighted research on medications, therapies, and diagnosing tools to treat myelofibrosis, hemophilia B, chronic graft-vs-host disease, necrotizing myopathy, and myeloproliferative neoplasms.
Read More
Report Highlights Case of Shellfish Allergy–Induced Overlap Chronic GVHD Following SCT
December 12th 2021In a recent case report, researchers outlined the case of a patients with shellfish allergy–induced overlap chronic graft versus host disease (ocGVHD)—a case of GVHD that has components of both acute and chronic GVHD.
Read More