A chronic, nonproductive cough is another major presenting symptom of patients with IPF. As many as 80% of patients with IPF experience chronic cough. Chronic cough usually lasts at least 8 weeks and ...

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 ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Miller, MD, a professor of pediatric endocrinology at the University of Florida in Gainesville who specializes in ...

Kine Sciences advanced KINE-101 to the phase 1b/2a study following the successful completion of a phase 1 study in the United States. The first patient in a new phase 1b/2a clinical trial to ...

The study found that 11.3% of the participants had a pathogenic or likely pathogenic variant. Furthermore, variants of uncertain significance were found in 32.5% of patients. Genomic sequencing for ...

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 ...

The prevalence of AATD in the offspring of parents with AATD in our study was 37%. A risk of pregnancy complications and adverse birth outcomes has been observed in mothers with alpha-1 antitrypsin ...

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 ...

The low positive predictive value indicates that a positive diagnosis using planar imaging would result in an incorrect diagnosis of ATTR-CM 42% of the time. Calculating visual scores using ...

Intravenously administered zaltenibart was shown to be safe and effective in phase 2 clinical trials. Enrollment is under way to recruit patients for a phase 3 study designed to assess intravascular ...

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 ...