Rare Disease Report®

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.

An ongoing assessment of caregivers considering hydroxyurea initiation for children with sickle cell disease provides a look into how such families are handling telemedicine and the pandemic.

Data show the average maximum cortical capillary RBC velocity is significantly greater in sickle cell mice compared to controls.

New ASH 2021 data shows how guidance is still lacking for clinicians responsible for addressing contraception with women with sickle cell disease.

This study evaluates the likelihood that young adults with sickle cell disease or sickle cell trait will have a child or expand their family.

The approval of daratumumab and hyaluronidase-fihj plus carfilzomib and dexamethasone is for treatment of adult patients with relapsed or refractory multiple myeloma who have received 1 to 3 prior lines of therapy.

There is currently not much known about the relationship between pregnancy outcomes and sickle cell trait.

There was no differences in complications by site in either the left or right subclavian vein.

In this video interview, Titilope Fasipe shares a summary of her plenary presentation on sickle cell disease from the National Organization for Rare Disease summit.

The agent is the first therapeutic treatment indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses.

Dr. Fasipe shares about her experience at the National Organization for Rare Disease Summit 2021 as the opening plenary speaker.

Hydroxyurea has been on the market for decades and yet it's underutilized as a treatment for sickle cell disease.

Ropeginterferon Alfa-2b-NJFT (Besremi) is approved by the FDA for the treatment of polycythemia vera regardless of a patient's treatment history.

Investigators also found a high prevalence between OSAS, nasal polyps, nutritional status, and other clinical variables.

As the opening plenary speaker at the National Organization for Rare Disease summit, Dr. Fasipe shares from her unique perspective as doctor and patient of sickle cell disease.

A cross-sectional analysis suggests children aged 5-7 years old may begin to reckon with the burdens of sickle cell disease, while toddlers and infants may not.

In the full interview with Dr. Fasipe, she challenges healthcare professionals to step up their understanding of sickle cell disease and the complications that patients are facing when they seek help.

ILD is a very broad category of chronic lung conditions with considerable nuance. Glassberg joins to discuss the roles of biomarkers, biopsies and drugs, as well as multidisciplinary clinics and stem cell therapy.

A sickle cell patient takes center stage to share with healthcare providers what it’s truly like living through a vaso-occlusive crisis.

In this video interview, Dr. Lee-Kim explains the importance of understanding the results of a Complete Blood Count before rushing to refer a patient to a hematologist.

Dr. Fasipe shares her in-person experience viewing Phillip Okwo's Life Interrupted production about living with sickle cell disease.

The approval is based on 24- and 80-week findings showing significant benefit in LDL-C reduction with the subcutaneous injection drug.