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 ...

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 ...

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 ...

Magnetic resonance imaging of the brain and spinal cord revealed lesions typical of patients with MS. The report highlighted the challenges in the diagnosis and management of this type of case, given ...

The overall survival rate at 18 months for patients diagnosed before the introduction of the new guideline was 79% while it was 90% after the introduction of the guideline. The number of patients ...