News

The historic phase 3 HOPE-B trial shows all but 1 treated patient discontinued prophylaxis after a single dose of etranacogene deaparvovec.

After a sudden reversal and complex analyses, aducanumab’s future rests in the hands of the FDA while the community stands split on if the available data are enough to justify an approval.

The second process of ARU-1801 in a new patient resulted in up to 4 times greater engraftment of transduced hematopoietic stem cells and progenitors at 6 months.

An interview with a Janssen expert on the growing portfolio and newest data for daratumumab.

The sickle cell disease subtype is associated with increased risk among patients as young as 2 years old.

In data presented at ASH 2020, FT-4204 shows early potential in being safe, efficacious treatment for sickle cell disease.

The ongoing phase I study also demonstrates that the drug is associated with an increase in Hb from baseline with a concomitant decrease in hemolytic markers.

Analyses of vital sign changes from the 6-minute walk test may be indicative of cardiopulmonary function and potential survival among patients with sickle cell disease.

The last episode of 2020 caps advances and ongoing research into the most burdensome chronic respiratory disease in the US.

A discussion on what the future of monitoring the rare disease may entail, and how COVID-19 interrupted in-person assessment earlier this year.

The combination therapy was linked to a record phase 3 progression-free survival rate among a subpopulation of patients with the rare cancer.

A discussion with a study author and longtime expert on improving monitoring of the rare disease.

The furthest-progressed gene-edited therapy for a genetic disease reported historic efficacy data in ongoing phase 1/2 trials.

A study finds that children with cognitive or emotional deficits trended towards an increased healthcare utilization.

Patients with baseline PROMIS-57 scores worse than population norms reported clinically significant improvements in 6 of 8 domains at 12 months post-treatment.

Patients with acute myeloid leukemia or myelodysplastic syndromes are at especially increased risk for increased disease severity and death.

New data from an ongoing trial showed patients achieved MRD negative status after a vaccine regimen—and have maintained status over a year.

Investigators evaluated patients from 3 Chicago hospital systems and assessed risk factors associated with thrombosis.

Early assessment from an Australian team of investigators is targeting a pathway linked to hemoglobin expression.

High levels of fetal hemoglobin have previously been linked to the mitigation of morbidity and mortality of sickle cell anemia.

Those without prior exposure to venetoclax experienced the most benefit.

Diabetic patients are more susceptible to severe COVID-19 outcomes.

Dr. Kausik Ray presents new data on inclisiran and bempedoic acid as cholesterol lowering medications.

This combination therapy may have a few advantages over standard corticosteroid regimens.

The indication is based on 48-week results showing significant benefit in reduced HAE attacks among pediatric and adult patients.

Patients with normal eGFR increased from 60% at baseline to 80% at 3 years following procedure.

Despite the difficult circumstances, experts believe colorectal cancer rates will not decrease in the coming years due to delays in screenings.

The top-line results from the ABX464 study conducted in 15 European countries, as well as the US and Canada, are expected during the second quarter of 2021.