Future Rare Diseases

Improvement in symptoms of Rett syndrome with trofinetide beyond clinical trial efficacy assessments: case reports

Pub Date : 2024-07-15 DOI: 10.1080/23995270.2024.2375191 Robin C. Ryther, Christine Palladino, Sanjeev V Kothare

Supporting positive patient experiences for rare disease care during disruptive times: findings from a national study

Pub Date : 2024-07-03 DOI: 10.1080/23995270.2024.2366153 S. Kellett, V. Tadic, H. Petrushkin, Jane Ashworth, Alan Connor, E. McLoone, Srilakshmi Sharma, E. Agorogiannis, Patrick Watts, Edward Hughes, Ailsa Ritchie, Rachel Pilling, J. Benzimra, Catherine Marsh, Daniel Pharoah, Jessy Choi, Andrew D Dick, J. Rahi, A. L. Solebo

Managing high blood sugar (hyperglycemia) during pasireotide treatment: A plain language summary of the B2219 study

Pub Date : 2024-03-19 DOI: 10.2217/frd-2023-0025 S. L. Samson, U. Feldt-Rasmussen, Shao-Ling Zhang, Yerong Yu, Przemysław Witek, Pramila Kalra, Marek Bolanowski