Rare Disease Report®

The sBLA for lanadelumab permits its use as a prophylaxis in children as young as 2 years old with the rare condition.

Individuals with sickle cell trait not only need to know their status to make informed family planning decisions, but also so they can watch out for the possible symptoms.

The mRNA vaccines BNT162b2 (Pfizer-BioNTech) and mRNA-1273 (Moderna) showed no risk of Guillain-Barré syndrome post-vaccination.

Dr. Carolyn Rowley explains, the whole reason for the Sickle Cell Trait Awareness Campaign (STAC) is to start talking about it.

"Despite advances in SCD management and high-risk pregnancy care, at the national level, outcomes in this population have not improved since the last NIS analysis of data from 1999 to 2008," investigators write.

Dr. Carolyn Rowley and Pat Corley, RN, explain why the new Sickle Cell Trait Awareness Campaign is designed for all middle school, high school, and university students.

Dr. Ian Flinn says many patients opt for treatment with Bruton's tyrosine kinase (BTK) over combination therapy.

The clinical trials that supported the FDA decision, SEQUOIA and ALPINE, evaluated the efficacy of zanubrutinib and findings paved the way to add the therapy as a treatment option.

The study population consists of patients who received 1-3 prior lines of therapy for relapsed and lenalidomide-refractory multiple myeloma.

The monoclonal antibody serves as the first and only therapy for treating hemolysis in adults with cold agglutinin disease, an extremely rare condition.

With public health efforts and therapeutic advancements the prognosis for this rare condition has progressed, but longer life expectancy has highlighted a multitude of areas that need attention in this population.

Dupilumab is currently the only treatment for eosinophilic esophagitis approved by the US Food and Drug Administration.

The FDA requested additional data showing the levels of a nitrosam impurity was below the set threshold throughout the life of vonoprazan.

Promising results from the IMerge phase 3 clinical trial met the primary and secondary endpoints in the investigation of imetelstat, a novel telomerase inhibitor developed for hematologic malignancies.

The topline phase 2b data shows both 75 mg and 150 mg doses of atacicept resulted in a 31% mean reduction in proteinuria.

The targeted patient population included those with sickle cell disease or beta thalassemia who were considered at high risk for complications, or otherwise ineligible for standard myeloablative hematopoietic stem cell transplant.

The results show similar rates o colonized C difficile among pediatric patients and adult patients with cystic fibrosis.

"The challenge right now is that we have no therapies in the emergency department to treat acute pain, outside of pain medications and IV opioids," Dr. Claudia Morris says in an interview.

The motive of the study wasn't to implement hematopoietic stem cell transplantation (HSCT) programs worldwide, but as a contribution to comprehensive cancer control efforts.

According to Dr. Corinna Schultz, a key part of the conversation of sickle cell trait (SCT) is centered around reproductive choices for the patient in the future.

The FDA's plans to expedite and expand regulatory pathways has resulted in a novel drug development boom since 2017. The commissioner discusses the details of their strategy.

Dr. Adam Lamble recommends that pediatric patients with AML and TP53 status should now be considered for transplant in first remission, and novel interventions are warranted.

In part A of the trial, 60% of patients treated with dupilumab weekly met the primary endpoint of histologic remission, compared to 5% of the placebo group.

Dr. Molly Tokaz and Dr. Lisa Force discuss the extensive data they collected on the utilization of hematopoietic stem cell transplantation (HSCT) for patients with AML.

Almost 25 years later, Dr. Claudia Morris concluded 2022 by presenting phase 2 data at the 64th ASH Annual Meeting that demonstrated the therapy's benefit, and her original premise. Her team's phase 3 trial evaluating Arg is currently underway.

Dr. Adam Lamble shares that outcomes for pediatric patients with acute myeloid leukemia that have TP53 status are inferior to other patients with high-risk disease.

Dr. Corinna Schultz talks about her latest data and the growing body of literature showing medical complications that can come with sickle cell trait.

"Using CRISPR/Cas9 you are able to actually fix a disease, that otherwise, was not fixable," Dr. Haydar Frangoul explains the data he presented at ASH 2022.

New developments in gene therapy offer a promising perspective of a future where curing sickle cell disease (SCD) is possible.

With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.