RDR Alert®

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.

This episode features a conversation with Dr. Amelle Shillington and Jacob and Ashley Wiley after Shillington diagnosed the extremely rare genetic disease that's afflicted Aislynn Wiley, and only 69 other documented cases worldwide.

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

Mehdi Mirsaeidi, MD, MPH, Director of the ILD and Sarcoidosis Program at the Jacksonville College of Medicine, joins Lungcast to review the rare disease for Sarcoid Awareness Month.

An overview of the progress in hemophilia treatments and global access to gene therapy.

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

Outside of the ankle, knees and elbows were also commonly affected joints for patients with hemophilia.

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

New data suggests that the half-life of nonacog alfa is longer than previously detailed in young and older patients.

The most common comorbidities for adults were hypertension, hyperlipidemia, and diabetes.

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

Positive preliminary biomarker data were reported from the first cohort in the phase 1 portion of the trial.

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

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

In an interview, Karen Kozarsky, CSO of SwanBio discusses the new data being presented at AAN on AAV-based gene therapy candidate SBT101.

At the AAN 2022 Annual Meeting, SwanBio presents new data regarding lead candidate SBT101 for treating adrenomyeloneuropathy, a rare debilitating disease with no approved therapies.

Dupilumab is the only biologic medicine to show positive, clinically meaningful phase 3 results in adults and adolescents 12 years and older with EoE.

The NaV1.8 inhibitor VX-548 shows promising potential as a non-opioid acute pain treatment.

Bristol Myers Squibb has to wait 3 months for FDA decision on Reblozyl for treating non-transfusion-dependent β-thalassemia.

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