Alltrna announces initiation of clinical trial with tRNA therapeutic AP003 Alltrna recently announced the initiation of the first clinical trial of a tRNA therapeutic, AP003. AP003 is designed to restore full-length protein production by inserting the correct amino acid at
Patient and Caregiver Experience with Reproductive Healthcare for Adolescents and Young Adults with Inherited Metabolic Disorders Patient and Caregiver Experience with Reproductive Healthcare for Adolescents and Young Adults with Inherited Metabolic DisordersThis research survey is being conducted by Dr. Jessica Gold from
OAA Connects with Metabolic Dietitians at GMDI 2026 Conference The Organic Acidemia Association (OAA) was proud to participate as an exhibitor at the recent Genetic Metabolic Dietitians International (GMDI) Conference, held April 23–25 at the Wyndham St. Louis. Representing OAA,
OAA is hosting a virtual town hall on May 31, 2026 at 6pm Central Time. Come to here about updates on our programming, exciting things to look forward to, and give feedback on what is important to you. Bring any
Gabriell, 26 years old, PA Hi all! My name is Gabriell Snyder. I’m 26 years old and was the first case of PA diagnosed through newborn screening in the state of Massachusetts. My first few years on this earth were
Organic Acidemia Genetic Testing 101 Genetic testing is a powerful tool for understanding inherited disorders. It can help clinicians diagnose a condition or confirm a diagnosis made with other tests. Patients with organic acidemias are often first identified through newborn
Eli, 4.5 years old, GA-1update In the Fall 2024 Newsletter, Mike and Sandra Wagner shared their story about their son, Eli. Diagnosed with GA1 through newborn screening, Eli suffered a metabolic crisis at just 5 months old. By the time
Emerald, GA-1 Update Emerald is a vibrant, determined three-year-old who happens to live with Glutaric acidemia type 1 (GA1), a rare metabolic disorder that requires careful, lifelong management. While her diagnosis shapes the structure of our days, it does not
Rare Disease Day 2026 Saturday, February 28, 2026 was Rare Disease Day. The OAA community shone a light on organic acidemias by showing up in stripes, OAA rare disease day shirts, and smiling faces.
Moderna mRNA clinical trial for PA update An update from Moderna on the clinical trial for an mRNA therapy for propionic acidemia.
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