I haven’t seen a pulmonologist in 5 years, and I would highly recommend that anyone with Pompe disease make it a priority. Rare Disease Advisor, a trusted source of medical news and feature content ...

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 ...

Researchers recently detailed the genetic and clinical features of congenital protein-losing enteropathy (PLE), with a focus on CHAPLE disease.

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 ...

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 ...

Several factors can contribute to the suppression of bone marrow erythropoiesis in infants with HDFN, including maternal antibodies, intrauterine transfusion, and simple transfusion. The suppression ...

The recommendations were based on the available evidence, its certainty, and key judgments using the Grading of Recommendations Assessment, Development and Evaluation framework. New guidelines ...

The guidelines highlight the unique needs of females and children, reflecting differences in disease presentation, triggers, and management. The Polish Society of Allergology has released updated 2025 ...

In CIDP, SOC therapies (immunoglobulins/corticosteroids) are burdensome, have variable efficacy, and significant side-effects. Two phase 3 clinical trials, MOBILIZE ...

The authors used individual patient data from garadacimab trials (phase 2 and 3 VANGUARD) and published summary data from the phase 3 HELP trial for lanadelumab. Garadacimab may offer superior ...

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 will measure hemoglobin levels among patients taking HRS-5965 who do not require transfusions during the study period. A new Chinese study is preparing to launch that will investigate the ...