The amyloid accumulation volume decreased significantly from 591.1 before tafamidis treatment to 213.8 following treatment. Tafamidis treatment inhibits the production of new amyloids in patients with ...

The diagnosis and management of CIDP are complex and best addressed by an interprofessional health care team that includes neurologists, neuromuscular specialists, physical medicine and rehabilitation ...

Larry Luxner, senior correspondent for Rare Disease Advisor, talks with Claire Booth, MBBS, PhD, of University College London in the UK and cofounder of the AGORA Initiative, which aims to tackle the ...

Taylor Kane is executive director of Remember the Girls, a nonprofit group that advocates on behalf of females with X-linked disorders (Photo courtesy of Taylor Kane) New Jersey native Taylor Kane ...

From landmark gene therapy approvals to illuminating discoveries into the pathogenesis of rare diseases, 2023 saw substantive advances in rare disease diagnosis and treatment. The following list of ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

Over 80% achieved a clinically meaningful improvement in at least one of the scales assessed. Treatment with risdiplam over 30 months led to overall clinically meaningful improvement in most adult ...

In addition to better efficacy and convenience, rFVIIIFc was found to be safe and well tolerated by all ages of patients with hemophilia A. Results of the European A-SURE study show that extended half ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

Efanesoctocog alfa improves hemostasis, reduces bleeding, and enhances quality of life for patients with severe hemophilia A, including those previously excluded from trials. Efanesoctocog alfa has ...

The rarity of generalized pustular psoriasis is associated with limited clinical experience and thus creates difficulty in the assessment of therapeutic proposals. The Psoriasis Working Group has ...

A new phase 2 clinical trial to test the safety, efficacy, and tolerability of TAK-411 for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) is now open. The open-label, ...