Methylmalonic Acidemia (MMA)
Methylmalonic acidemia, or MMA, is a rare condition that affects how the body breaks down food. It can cause serious problems like seizures and delays in growing and learning. Right now, MMA is treated by eating less protein, taking special vitamins or antibiotics, or sometimes getting an organ transplant.
Diagnosing MMA
MMA is often detected through routine newborn screening shortly after birth. It’s a genetic disorder, meaning it’s passed down through families. Some people may be tested because of family history, while others may not be tested and diagnosed until symptoms arise.
The most common type of MMA happens because of changes in a gene called the MUT gene. This gene helps make the MUT enzyme, which is important for breaking down certain proteins and fats. Without this enzyme, the body struggles to turn food into energy, and harmful substances can build up in the blood. test
Symptoms
Individuals with MMA may experience a range of symptoms that can vary in severity. These include:
Poor Feeding and Loss of Appetite
Individuals with MMA can’t convert food into energy, so they miss out on essential protein and fat needed for proper growth.
Fatigue
Individuals may experience low energy levels and excessive tiredness.
Developmental Delays
MMA can cause neurological complications, such as seizures, movement disorders, developmental delays, or coma.
Vomiting
MMA can cause vomiting in individuals, which in turn may lead to dehydration.
Poor Muscle Growth
Low muscle tone, leading to reduced muscle strength in the body.
Landmark Study
The Landmark Study is evaluating if an investigational treatment is safe and can impact blood levels of substances known as biomarkers, which may help measure function of the MUT enzyme in participants with MMA. mRNA-3705 is an investigational intravenous (IV) infusion treatment that is intended to instruct a person’s body to make the MUT enzyme work better.
To learn more about any of our rare disease clinical trials or to see if you may qualify to join, please contact the Moderna WeCare Team.
All investigational medications and treatments must go through several stages of research (also called “phases of research”) to determine if it is safe and effective. Clinical trials are usually conducted in 4 phases that build on one another, and each phase is designed to answer certain questions in an ethical manner.
- Phase 1: A trial of a small number of participants to find safe doses, gather information about how the investigational treatment interacts with the human body, and look for side effects.
- Phase 2: A trial of more participants to further evaluate how the investigational treatment affects the body, gather safety data, and monitor side effects.
- Phase 3: A trial with the largest number of participants to gather more safety data, see if the investigational treatment is effective, and compare it to existing treatments.
- Regulatory Approval: A regulatory agency reviews data from the clinical trial and determines if the investigational treatment is safe and effective for use in individuals with the condition.
- Phase 4: After receiving regulatory approval, the investigational treatment is made available to the public. This phase of research is used to continue to monitor the safety of the treatment, even after it has been approved for public use.
Children are not small adults. Adult treatments may not always work the same way in children, or some treatments may be better suited for children. Some conditions affect only children, so it is important for researchers to understand the safest and most effective dose of medication for children across all age ranges.
Yes, participation in any clinical trial is voluntary. Participants are free to discuss any questions with the trial doctor or trial team at any time. Participants also have the right to withdraw from the PAthway Trial at any time and for any reason.
Participants will receive compensation for their time as well as reimbursement for travel and other trial-related expenses.
For certain clinical trials, please contact Moderna to find out if your trial offers compensation.
