Encouraging Progress in Fibrosis Research

Rare Disease Day 2025 is the perfect day to see an announcement on the new advancements in understanding and treating fibrosis. A new initiative led by our Scientific Advisory Board member, Dr. Maria Macías, and her team at IRB Barcelona is focusing on developing potential therapies for fibrosis-related diseases like Myhre syndrome. Their work is centered on identifying innovative treatment strategies that target key molecular mechanisms involved in disease progression.

Dr. Macías’ research explores how specific proteins in the SMAD family influence fibrosis development. Her team is currently working on an initiative aimed at creating a new class of drugs designed to slow the progression of fibrosis. By refining drug design through structural analysis, they hope to develop a more precise and effective approach to managing Myhre syndrome.

While this research holds significant promise for Myhre syndrome, it also has wider implications for conditions such as idiopathic pulmonary fibrosis and cardiac fibrosis. Developing targeted treatments could lead to advancements that benefit a broad range of patients with fibrosis-related organ failure.

We remain committed to championing research that can lead to meaningful medical breakthroughs and are collaborating with Maria Macias and IRB Barcelona for our preclinical projects. We look forward to continued progress in developing therapies that can improve the outcomes for Myhre syndrome patients worldwide.

This initiative is supported through funding from the Agency for Management of University and Research Grants (AGAUR). We extend our appreciation to the dedicated researchers and supporters working toward innovative solutions for fibrosis-related diseases.

Read the full press release and see a video from IRB Barcelona here