As discussed at the 2016 MSUD Symposium and in the previous issue of this newsletter, Acer Therapeutics is following up on the research initiated by Dr. Lee on phenylbutyrate and its potential to aid in the control of branched chain amino acid levels in those with MSUD. I was asked to provide a quick update on ACER-001 – the taste-masked, immediate release formulation of sodium phenylbutyrate – that we at Acer are developing for the treatment of MSUD.

We’ve spent the better part of the past year optimizing the formulation, ensuring that it is completely taste-masked, yet behaves like regular sodium phenylbutyrate once it’s in the body. It’s really important that we get this right, so that patients taking ACER- 001 get the maximum benefit of the drug, while not detecting the horrific taste of phenylbutyrate! While this has been an incredibly challenging project, we believe we now have the best formulation developed to take into the clinic.

Before we begin the phase 2 clinical trial for MSUD, we need to run a quick study in healthy volunteers to make sure the drug is completely taste-masked, safe, and behaves similarly to regular phenylbutyrate in the body. This is a very quick study that should be completed in the fall. Assuming everything goes well with this study, we will begin the MSUD trial shortly afterward.

As a reminder, the MSUD phase 2 trial will enroll approximately 60 patients at 10-15 clinics across the US. Once a patient begins the study, they will be required to maintain a very tightly-controlled diet for about a month. ACER-001 will be added on top of their diet for about two weeks, to determine if ACER- 001 + diet can lower levels of leucine by a meaningful amount over diet alone.

We invite you to visit our recently updated website, where you can access patient resources, subscribe to our mailing list to receive program updates and clinical trial information, or ask questions directly to the company. Please visit:

We look forward to hearing from you, and keeping you updated on our progress!

Melvin Carruth

This letter is a tribute to my brother Melvin Carruth! We believe he is one of the oldest living African Americans with Maple Syrup Urine Disease.

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NBS-MSUD Connect: Your One-Stop Shop For MSUD Resources

NBS-MSUD Connect was launched as part of the Newborn Screening Connect patient registry (NBS Connect) in 2013 through a partnership between the Department of Human Genetics at Emory University, the Maple Syrup Urine Disease (MSUD) Family Support Group and other key stakeholders.

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Cambrooke Therapeutics

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Emory Metabolic Camp 2018 Announcement

Join us June 18-23, 2018 for the 24th Annual Metabolic Camp at Emory University in Atlanta, GA!

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From The Chairman’s Desk

As I sit here reflecting on how much the care and treatment of MSUD has changed over the years, my mind goes back to 1978 when our son Keith was born.

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MSUD Advocacy Report

Medical Nutrition Equity Act

The Medical Nutrition Equity Act (MNEA) would require all private insurance plans (state regulated or self-insured/self-funded) and federal health programs, including Children’s Health Insurance Program, Tricare, Medicaid, Medicare, and Federal Employee Health Benefit Plans, to provide coverage for formula and low-protein foods for all children and adults with MSUD.

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The MSUD Family Support Group Is Excited To Announce Their Participation In The Million Dollar Bike Ride

The MSUD Family Support Group is excited to announce their participation in The Million Dollar Bike Ride

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19th Biennial MSUD Symposium

I can’t believe that almost two years have passed and it is time for another MSUD Symposium! I am especially excited about this conference because I’ve built in extra time for social interaction.

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A Child's Life

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