Fingolimod treatment leads to beneficial effects in Huntington disease (HD), according to a new study in a mouse model of the ...

Vosoritide was approved in 2021, after current achondroplasia guidelines were published, creating a need for treatment and monitoring recommendations. A new international consensus statement has been ...

There were fewer multiorgan transplants in patients with hereditary ATTR-CM with increased use of disease-modifying therapies, a study found.

Overall, 80.9% of patients were in complete remission 1 year after diagnosis irrespective of GPA or MPA cases (79.7% vs 83.8%, respectively). The therapeutic armamentarium for individuals with ...

The navenibart phase 3 program will consist of the ALPHA-ORBIT phase 3 trial and the long-term extension trial, which are designed to support registration globally. Global start-up activities ...

Surgery is not often used as a treatment option for chronic lymphocytic leukemia (CLL) because the disease is usually widely spread in the bone marrow and other organs. However, surgery may sometimes ...

Vamorolone at a dose of 2 mg/kg a day was associated with improvements in the velocity of the 4-stair climb test and the supine-to-stand test. A new systematic review and meta-analysis of the ...

Nusinersen, an SMN2-targeted antisense oligonucleotide drug administered by intrathecal injection, was able to improve motor function and survival. Treatment with nusinersen can improve motor function ...

Dr. Bichell is part of a panel studying how the US FDA can speed up clinical trials and be more transparent about its decisions. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...

rhG-CSF has a short half-life and requires daily injection to maintain an effective blood concentration. Pegylated recombinant human granulocyte colony-stimulating factor (PEG-rhG-CSF) reduced the ...

The adolescent HAE population continue to experience unique challenges to their school and social lives that degrade their health-related quality of life. Adolescents with hereditary angioedema (HAE) ...

It is important to include IgG4-RD in the differential diagnosis when investigating other immune-related conditions. An elderly man with constrictive pericarditis was diagnosed with immunoglobulin ...