Rare Blood

Case Study: Caplacizumab May Salvage Refractory Immune TTP Following COVID-19 Vaccine

May 20th 2022, 4:08pm


While suggesting that the disorder be included in the differential diagnosis of thrombocytopenia following vaccination, the group emphasized that the benefits of global vaccination against the virus outweigh the risk of this rare complication.

Mortality Risk Appears to Spike in Winter Among Patients With Cold Agglutinin Disease

May 18th 2022, 10:52am


Patients with cold agglutinin disease faced a 4.5-times greater risk of death during winter months, new data show.

Montreal Cognitive Assessment May Hold Promise for Assessing Cognitive Impairment in SCD

May 14th 2022, 11:05am


Among their sample cohort of 49 patients, the researchers found that scores on the Montreal Cognitive Assessment correlated with measures of both literacy and health literacy.

Walgreens’ Ray Tancredi Weighs in on Expensive Hemophilia Gene Therapies in the Pipeline

May 13th 2022, 5:59pm


Gene therapies in the pipeline to treat hemophilia will be very expensive, but they also seem to be very efficacious, and multiple could hit the market between now and 2024, said Ray Tancredi, RPh, MBA, CSP, divisional vice president, specialty pharmacy development and brand Rx/vaccine purchasing, Walgreens.

Adolescent, Young Adult Males With Sickle Cell Disease Lack Knowledge of Fertility Implications, Study Shows

May 12th 2022, 4:19pm


Improved management of sickle cell disease has vastly improved life expectancy and reduced morbidity for affected patients, but the disease itself and its treatments carry fertility risks that many young patients and their caregivers are unaware of.

Cancer Treatments, Orphan Drugs, Gene Therapies Represent Big Areas to Watch in Specialty Drug Pipeline

October 20th 2021, 8:30pm


During her presentation at the Academy of Managed Care Pharmacy Nexus 2021 meeting, Aimee Tharaldson, PhD, highlighted the most anticipated specialty pharmacy drugs awaiting FDA approval, including several treatments for cancer and rare diseases.

Gene Therapy for Hemophilia B Shows Positive Results in Phase 3 Trial

June 25th 2021, 1:30pm


The company, uniQure, said it is the largest group of patients with hemophilia B to receive a single gene therapy investigational product so far. 

Gene Transfer Therapy Requires Further Investigation Despite Potential Approval, Review Says

April 23rd 2021, 8:17pm


After a more than 35-year pursuit, the licensing of a gene therapy product for treating hemophilia may be available within 2 years.

Study Finds Inhibitors Develop in Nonsevere Hemophilia A Earlier Than Previously Reported

October 30th 2020, 8:09pm


Up until now, it was thought most patients with hemophilia A developed inhibitors in middle-age. A new study suggests it usually happens in the early teens.