Rare Disease Report®

In this interview, Valton details the editing function of TALGlobin01 and how it stands out among traditional gene therapy approaches to sickle cell disease.

Björn Mellgård gets technical as he describes the function of TAK-755, the recombinant ADAMTS13 enzyme, in sickle cell disease and cTTP.

Investigators developed the first module to confront implicit bias toward patients with sickle cell disease on an individual or personal level.

The American Society of Hematology debuted its Health Equity Rounds series, which are interactive, case-based discussions with an interdisciplinary panel to include hematologists, health equity/public health experts, patients, and patient advocates.

The clinical program has been suspended for patients that are under the age of 18 after an adolescent patient developed persistent anemia.

Fuad El Rassi talks about the Grady Comprehensive Sickle Cell center, the COVID-19 vaccine, and the data he presented at ASH.

The treatment represents the first approved treatment that directly targets sickle hemoglobin polymerization.

Findings from the SWAY trial highlight a pressing need for varying and more personalized treatment methods for patients.

TAK-755, a recombinant ADAMTS13, is examined for its therapeutic benefit to patients with sickle cell disease and patients with cTTP.

The treatment resulted in a significant reduction of proteinuria from baseline in 34% of trial participants.

The approval represents the first ever treatment for the disease in patients aged 2 years and older.

The enrollment distribution was consistent by ethnicity over the course of 18 years, with no change in diversity over time despite there being an increase in the UK in the minority population.

Of the SCD patients who contracted COVID-19, 49% of them visited the Grady Comprehensive Sickle Cell center because of a vaso-occlusive event.

Data show patients with sickle cell disease identified by distinct cognitive profile should have neurorehabilitation tailored to each unique profile.

After 2 months of voxelotor treatment, all patients with sickle cell anemia perceived global improvement, but peak VO2 did not change in 8 of 9 patients.

Data show most patients (60.3%) with sickle cell disease achieved a Hb increase of >1 g/dL during the follow-up period.

Patients with SCD were not at an increased risk of death or disease complications.

High dose vitamin D supplementation has been linked to fewer pain days and higher physical activity scores for patients with chronic pain.

New ASH 2021 data for the stem cell transplant therapy suggest it has a durable and safe benefit for adults with severe disease.

Investigators added improvements were needed regarding access to complete diagnostic evaluation among Black patients, citing disparities in care.

Data show the majority of patients who initiated crizanlizumab remained on therapy, while a third of patients saw barriers including insurance coverage.

New research on the preclinical product TALGlobin01 reveals the potential impact that gene therapy can have on the future of sickle cell disease treatment.

Investigators aimed to improve lentiviral vectors to boost therapeutic β-like globin levels without increasing the mutagenic vector load in hematopoietic stem/progenitor cells.

New data for the Rocket Pharmaceutical gene therapy candidate show durable benefit in patients 6 months out from transplant.

The global biopharmaceutical company released research data on rozanolixizumab and zilucoplan at the 2021 ASH annual meeting and exposition.

An international assessment shows many patients are wary of emergency rooms, and clinicians want more diverse therapies.

Data show 12 of 25 strokes (48%) in a cohort of children did not met the definition of typical stroke related to sickle cell cerebral vasculopathy.

Defined roles and research-informed recommendations are lacking for clinicians navigating birth control with females with sickle cell.

Patients with sickle cell disease often suffer from acute kidney injuries and chronic kidney disease.

Shaji Kumar, MD, presents final Phase 3 data of the BELLINI trial at the ASH 2021 conference.