RDR Alert®

The aim is for the RNA-based medicine to dually serve as a treatment for multiple forms of transthyretin amyloidosis, and as a commercial contender to competing therapies.

In honor of World Sickle Cell Day, Dr. Titi Fasipe shared a comprehensive overview of the current status of gene therapy and the techniques being used for sickle cell disease treatment.

New research from the UK suggests educational tools for caregivers are in use and beneficial, but none are universally applied.

The survey identified long-term health complications of sickle cell disease as a primary concern among patients and providers and overall understanding of the disease needs more educational support.

There is not much known on the link between sickle cell disease and romantic relationships, particularly from the male perspective.

Dr. Temeia Martin talks about how her team identified the transition period as a major obstacle for patients with sickle cell disease and what her task force has implemented as a solution.

An increased risk of obesity and decreased risk of abnormal weight loss was also observed in patients born to mothers with sickle cell disease.

With World Sickle Cell Day approaching, Dr. Titi Fasipe reflects on the developments within the field, such as the continued conversation of disparities and the increased comfort level with new therapies.

Vutrisiran is the first and only FDA-approved treatment that’s demonstrated reversal in neuropathy impairment with subcutaneous administration once every 3 months.

Some of the favorable responses to voxelotor treatment and signs of hematologic response after voxelotor treatment included increased Hb levels, decreased reticulocyte percentage, and decreased total bilirubin.

Intravenous opioid use for VOC decreased by 52% in inpatient services between 2017-2018, while oral opioid use increased.

Currently many more patients undergo right heart catheterization than is necessary for diagnosing pulmonary hypertension in patients with systemic sclerosis.

There is a growing body of evidence showing the value of rituximab in the treatment of SSc-ILD.

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

Efanescotocog alfa is the first factor VIII therapy to be granted Breakthrough Therapy designation by the FDA.

New information suggests a few more cases have surfaced in the United States, pending CDC confirmation.

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).

Sarcoidosis expert Dr. Wonder Drake got into the field after diagnosing a patient with the disease early in her career. In this interview she explains the best way to approach a suspected case of sarcoidosis.

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.

In this interview, Dr. Wonder Drake shares physician resources and explains the unique presentation of sarcoidosis and how it varies by region.

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

Isatuximab in combination with carfilzomib and dexamethasone (Kd) demonstrate the longest median progression free survival (mPFS) on a proteasome inhibitor backbone in multiple myeloma patients who relapsed after a prior therapy, including lenalidomide.

With this new decade-long cohort, Dr. Chen notes that researchers are "one step closer to a more accurate view of what sarcoidosis may be like in the United States", with has been suggested to be more prominent in African American communities.

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.

ANCA-associated vasculitis may present like sinusitis, but it typically affects multiple organ systems.

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