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
On Rare Disease Day, PKD Foundation, IQVIA Highlight Patient Registry Gathering Quality-of-Life Data
February 28th 2023The Polycystic Kidney Disease Foundation and IQVIA are partners in the development of the ADPKD Registry, which is tracking longitudinal quality-of-life information from patients across North America.
Read More
Similar Survival Rates Seen Among Black African Americans With PMF, Other Groups
February 23rd 2023The study also found, however, that transplant outcomes appear to be worse for Black African American patients who have primary myelofibrosis (PMF), although the study utilized a small sample size.
Read More
Spleen Response Closely Linked With Survival in MF While on Ruxolitinib
February 20th 2023Patients with myelofibrosis (MF) who achieved a spleen response while taking ruxolitinib and lost it had survival outcomes similar to patients who did not respond, according to results from a real-world study.
Read More
Genetic Screening Could Have Promise for Reducing Infant Deaths
February 11th 2023An analysis of 112 infant deaths found that the contribution of genetic diseases to mortality was higher than previously known, and that treatments are available for 30% of the genetic diseases uncovered by the study.
Read More
Top 5 Most-Read Rare Disease Articles of 2022
December 23rd 2022The most-read rare disease articles of the year included the topics of new pediatric central nervous system tumor guidelines, graft versus host disease, the contribution of gene variants to pediatric neuropsychiatric conditions, and hope for patients with blast-phase chronic myeloid leukemia.
Read More
Need for More Research Into Chronic GVHD After Allogeneic HSCT Highlighted in New Review
November 9th 2022Although the understanding of chronic graft-versus-host disease (GVHD) has improved in recent years, more work is needed to facilitate personalized treatments for patients after hematopoietic stem cell transplantation (HSCT).
Read More
Non–Driver Gene Mutations May Hold Predictive Value in ET and PV
November 1st 2022A recent study supports the notion that non–driver mutations in essential thrombocythemia (ET) and polycythemia vera (PV) have predictive value and found that a proposed international prognostication model may be useful for Japanese patients.
Read More
Stem Cells Highlighted as Potential Therapeutic Option in Huntington Disease
October 15th 2022Although no pharmacological treatment offers long-term efficacy or extends survival with Huntington disease at this point, research suggests that stem-cell therapy holds promise in this and other neurodegenerative diseases.
Read More
Patients With Lower-Risk MF, Low- or High-Risk ET Report Significant QOL Impacts
October 12th 2022Despite the use of therapies to address the clinical manifestations of myelofibrosis (MF) and essential thrombocythemia (ET), the symptom burdens of these conditions continue to negatively affect patient quality of life (QOL).
Read More
A Texas judge ruled that HIV pre-exposure prophylaxis (PrEP) coverage under the Affordable Care Act is unconstitutional; an FDA advisory panel recommended the approval of an experimental drug for amyotrophic lateral sclerosis (ALS); Walmart and UnitedHealth Group are collaborating to provide preventive health care for older Americans.
Read More
Guidelines Needed for Managing MPNs in AYA Patients, Review Says
September 5th 2022The management of myeloproliferative neoplasms (MPNs) in adolescent and young adult (AYA) patients is currently similar to that of older patients. However, comprehensive research is needed to form guidelines for this younger population.
Read More
Olipudase alfa (Xenpozyme) was granted FDA approval for patients with Acid Sphingomyelinase Deficiency (ASMD); Moderna and Pfizer-BioNTech’s bivalent COVID-19 vaccines are each authorized for use as a booster dose; people with type A blood have an 18% higher risk of stroke before age 60, compared with other blood types.
Read More