Sickle Cell

The agency also granted rare pediatric disease designations for inclacumab and GBT601.

In a late-breaking presentation at ASGCT 2022, investigators shared preclinical results of a gene-editing strategy that seeks to reactivate developmentally silenced fetal hemoglobin (HbF, α2γ2) in order to replace defective sickle hemoglobin (HbS, α2βS2).

Dr. Carolyn Rowley talks about the Cayenne Wellness Center's mental health initiative for individuals with sickle cell disease and shares the story of one of her first patients.

Sickle cell warriors are empowered to be part of the research and to be part of the solution, Regina Hartfield says.

Investigators who say that assessing vaso-occlusive crises solely through medical utilization doesn’t provide a full picture of the SCD experience because many VOCs are managed from home, are now backed by research.

Dr. Carolyn Rowley is leading the California Sickle Cell Disease Mental Health and Wellness Initiative to provide mental health services free to people living with sickle cell disease.

President and CEO of SCDAA Regina Hartfield discusses how the association will use the funds from the leadership award.

Investigators observed a potential risk between Black individuals with baseline signs of dehydration, SCT and related mortality over 30 years.

Voxelotor is the first and only FDA-approved medication for children with sickle cell disease.

The investigative gene therapy achieved significant phase 3 endpoints, after receiving feedback from the FDA on its efficacy outcomes in 2020.

A systematic literature review highlighted key risk factors that could constitute a risk assessment model for hemophilia bleeding—a currently lacking guidance for clinicians.

Concluding their discussion on sickle cell disease management, expert panelists share key unmet needs and hopes for the future.

A new study evaluates the prognostic potential of exercise testing for patients with sickle cell disease.

A study highlights methods to define hydroxyurea exposure as means of understanding hematologic benefit derived from the first-line drug for children.

Shared insight on patient education surrounding gene therapy in sickle cell disease and common misconceptions about this approach.

An overview of gene therapy in sickle cell disease with a focus on the risk-benefit ratio observed in clinical trials and practice.

Barriers to sickle cell disease care are identified across multiple levels including individual, family, provider, and socio-environmental levels according to patient perspectives.

Panelists take a step back to consider when they might use certain disease-modifying therapy in the setting of sickle cell disease.

Considerations for voxelotor’s unique mechanism of action and prospective use in patients with sickle cell disease.

Interstitial lung disease expert explains the importance of educating patients with autoimmune disease.

Panelists share their perspectives on the selection and use of crizanlizumab in certain patients with sickle cell disease.

Shared insight on L-glutamine therapy in sickle cell disease, with deference to observed efficacy and safety considerations.

Patients with Fabry disease who received treatment with PRX-102 every 4 weeks displayed a stable condition at the end of the trial.

Expert perspectives on hydroxyurea as a staple therapy in the treatment armamentarium for sickle cell disease.

Focusing on disease-modifying therapeutics, expert panelists discuss foundational treatment goals and guidelines for sickle cell disease.