Future rare diseases

Plain language summary: treatment of paroxysmal nocturnal hemoglobinuria with pegcetacoplan for 48 weeks (PEGASUS study)

Pub Date : 2024-05-05 DOI: 10.2217/ifrd-2023-0018 R. D. de Latour, Jeff Szer, I. Weitz, Alexander Röth, B. Höchsmann, Jens Panse, Kensuke Usuki, Morag Griffin, J. Kiladjian, Carlos de Castro, Hisakazu Nishimori, M. Al-Adhami, Pascal Deschatelets, Cedric Francois, A. Risitano, Peter Hillmen

What is the impact of long-chain fatty acid oxidation disorders on everyday life? Plain language summary of a survey

Pub Date : 2024-04-22 DOI: 10.2217/frd-2023-0030 Christy Abrams, Kristin Voorhees, Eliza Kruger

The effects of garetosmab in people with fibrodysplasia ossificans progressiva (FOP): a plain language summary of the LUMINA-1 study

Pub Date : 2024-04-11 DOI: 10.2217/frd-2023-0028 Maja Di Rocco, Robert J Pignolo, Richard Keen, Dushyanth Srinivasan, Scott J Mellis, Michelle Davis, Philippe Orcel, Christian Roux, Małgorzata Szczepanek, J. Bachiller-Corral, Angela M Cheung, Kathryn M Dahir, M. Mukaddam, Anita Boyapati, Kusha A Mohammadi, Aris N Economides, Robert J Sanchez, Dinko González Trotter, Susan Rhee, Gary A Herman, R. DelGizzi, George D Yancopoulos, E Marelise W Eekhoff, Frederick S Kaplan