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

Alpha-1 antitrypsin deficiency (AATD) is found to be associated with pregnancy-related complications and adverse birth ...

ATTR-PN diagnosis requires a combination of neurological and non-neurological tests, such as blood pressure monitoring, a ...

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 ...

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 ...

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 ...

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) ...

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 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 ...

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 ...