The studies indicated comparable growth trends regardless of whether therapy was initiated before or after age 5. Vosoritide, a once-daily injectable therapy, may improve growth outcomes in children ...

The US Food and Drug Administration (FDA) approved Panzyga, manufactured by Octapharma AG, for the treatment of PI and ITP in 2018 and for CIDP in adults in 2021. Octapharma commercializes Panzyga ...

The severity of clinical manifestations in CAD can vary depending on the specific type of cold-sensitive antibodies involved. While cold agglutinin and Donath-Landsteiner antibodies recognize antigens ...

Patients with CLL and a CSHA CFS score greater than 3 had significantly higher rates of treatment interruption due to adverse effects. The CSHA Clinical Frailty Scale (CFS) is a valuable tool for ...

IgG4-RD can mimic other medical conditions, including labyrinthitis, as demonstrated in this case report. A middle-aged patient was diagnosed with immunoglobulin G4-related disease (IgG4-RD) affecting ...

For the last 3 years, the PBC Foundation, based in Edinburgh, Scotland, has conducted an annual summit that addresses the challenges and potential remedies for those living with primary biliary ...

Dan Lier has light chain (AL) amyloidosis—the most common form of amyloidosis. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews former college basketball player Dan Lier, a ...

The patient recovered from surgery with no respiratory complications or any exacerbation in the symptoms of CIDP. Researchers from Iran presented the case of a patient with chronic inflammatory ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

The overall pooled prevalence of cirrhosis in individuals with homozygous ZZ AATD was 8.17%. Individuals with homozygous ZZ alpha-1 antitrypsin deficiency (AATD) face a substantial burden of liver ...

Results showed a significantly higher prevalence of liver disease among patients with chronic pruritus compared to healthy controls. Patients with liver diseases of different etiologies, including ...

Yiannis Papazoglou is one of only 40 people in the world with a mutation of the IRF2BPL gene. Foundation Aims to Help Patients With Rare Diseases—Even Before They’re Recognized The Undiagnosed ...