The study provides the strongest evidence to date supporting the potential of proximal pathway inhibitors to enhance clinical outcomes and quality of life for patients with PNH. Proximal pathway ...

Factor-H works to alleviate extreme poverty among people with Huntington disease in Venezuela, Colombia, and Peru. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ignacio Muñoz ...

Danicopan is being developed to enhance complement C5 inhibitor therapy, like eculizumab and ravulizumab, by selectively inhibiting factor D within the complement system. Among patients with ...

SGLT2i therapy was significantly associated with lower mortality but the association was no longer statistically significant when potential immortal time bias was considered. Treatment with ...

(Getty Images) Holistic approaches for people with DMD can include occupational therapy and personalized nutrition. While researchers continue to search for a cure for DMD, steps can be taken to help ...

Participants will receive subcutaneous doses of RLYB212 every 4 weeks from gestational week 16 until delivery. The European Medicines Agency (EMA) and the UK’s Medicines and Healthcare products ...

Left ventricular dilatation was present in 43% of the patients and 10% of patients had both a left ventricular ejection fraction of less than 50% and severe left ventricular dilatation. There is ...

Third-trimester sFlt-1 levels offer insight into neonatal risk in pregnancies with HPA-1a alloimmunization, potentially guiding intervention decisions in FNAIT. An association between elevated ...

The valuation of the hypothetical treatments identified the burden of treatment administration as the driving force of the ranking of treatments. A reduced risk of severe adverse events and the need ...

Having somebody to talk to, a person who understands what it’s like living with myasthenia gravis (MG), or another rare disease, is difficult to find. We all wish we had that person to speak with ...

Artificial intelligence may help physicians better predict infant diseases and introduce necessary interventions in a timely manner. Artificial intelligence (AI) is a powerful tool that can be ...

The Supplemental Oxygen Access Reform (SOAR) Act is stalled in the US Congress. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Amy Case, MD, chief medical officer of the ...