Rare Disease Report®

FDA Approves First Ever Biologic for Hypereosinophilic Syndrome

September 25, 2020

Mepolizumab will be used to treat both adult and pediatric patients with HES for at least 6 months without an identifiable non-hematologic secondary cause.

Unintentional Nonadherence to Hydroxyurea Prevalent Among Sickle Cell Patients

September 25, 2020

A study found that patients more commonly self-reported unintentional barriers to treatment adherence—such as forgetfulness and external influences—than intentional ones.

Pepinemab Yields Cognitive Benefit in Huntington Disease Patients

September 25, 2020

The treatment could also be used for Alzheimer disease and mid-stage Huntington disease for patients who suffer from greater cognitive deficits.

Sickle Cell Anemia Associated with Leg Length Discrepancies Among Adolescents

September 24, 2020

These discrepancies are more pronounced in young, adolescent females and older adolescent males.

Challenges Remain for Gene Therapies Targeting Hemoglobinopathies

September 22, 2020

Gene addition and editing strategies have potential of curing sickle cell disease and transfusion-dependent thalassemia, but there are still many obstacles that must be overcome.

Sickle Cell Disease Patients Lack Education on Transfusions

September 17, 2020

Interview results show patients can generally recall giving transfusion consent, but not information on the procedures benefits and risks.

Findings Uncover Disparity in Alpha-Thalassemia Screening Procedures in Newborns

September 17, 2020

The methodologies used to screen sickle cell disease are also able to detect alpha-thalassemia, which is currently not a core condition of the United States Recommended Uniform Screening Panel.

FDA Approves New Acute Myeloid Leukemia Treatment

September 01, 2020

Azacytidine 300 mg tablets, CC-486 is earmarked for AML patients who achieved their first complete remission following intensive induction chemotherapy.

FDA Issues CRL for Hemophilia A Gene Therapy

August 19, 2020

Valoctocogene roxaparvovec would be the first ever approved gene therapy to treat patients with hemophilia A.