Explore the Latest in Research, Trials & Treatment Advancements

Stay informed about the latest developments in research, trials, and treatment advancements in the field of rare inborn errors of protein metabolism. Living with a rare condition presents unique challenges, but ongoing research offers hope through emerging treatment pipelines for various IEMs. For inquiries regarding trials and research, please reach out to our dedicated MDDA Patient Pathways Nurse.

Latest Research Updates

This list below highlights key areas of ongoing research but is not exhaustive. Some of these studies may have published results. For independent and credible information on clinical research, peer-reviewed scientific journals are the most reliable source.

Clinicaltrials.gov – a site for approved (those with ethics approval) research trials lists 5 MSUD studies

There are no clinical treatments trials currently listed for Tyrosinemia but there are several papers on potential treatments in early stages of development:

Clinicaltrials.gov – a site for approved (those with ethics approval) research trials lists 5 HCU studies

Our Commitment to Keeping You Informed

At MDDA, we are dedicated to staying up to date with the latest advancements in treatments and research for all IEpMs. We regularly update this page to provide you with the most current and accurate information available.