Dr. Dwi Kemaladewi has played a key role in developing a novel therapeutic approach, that has opened new treatment avenues for MDC1A patients. Her team’s work now involves improving the effectiveness and safety of this technology even further. Dr. Kemaladewi is part of a research team that has developed a novel therapeutic strategy which resulted…
Steven Axon is the CEO of Prothelia, a biopharmaceutical company based in Massachusetts, USA, committed to developing a promising new treatment for MDC1A patients. Patients with MDC1A have a mutation in the LAMA2 gene – a gene responsible for making a protein called laminin-211. In the body, muscle cells and muscle fibres are surrounded by…
The Congenital Muscle Disease International Registry (CMDIR) aims to advance neuromuscular research by collecting vital data and by growing advocacy for congenital muscular dystrophies. The CMDIR was created in 2009 with the aims to raise awareness and standards of care, assist patients with enrolment in clinical trials and generally aid the research community in developing…
