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

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 ...

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

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 ...

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 ...

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) ...

Ocular manifestations of MPA are uncommon, but early recognition may allow for prompt initiation of appropriate treatment to prevent further complications. An elderly man with retinal vasculitis was ...

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 ...

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 ...

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 ...

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 ...

Patients with ANCA-positive interstitial lung disease (ILD) exhibited a high prevalence of fibrotic patterns and mortality largely driven by the ILD itself. In patients with interstitial lung disease ...