Organic acidemias (MMA, PA, IVA, GA1)
Autosomal recessive disorders of amino acid catabolism — methylmalonic acidemia (MUT, CblA/B/D), propionic acidemia (PCCA/PCCB), isovaleric acidemia (IVD), and glutaric aciduria type 1 (GCDH) — where toxic organic acids accumulate when protein catabolism outpaces the residual enzyme. Classical forms present neonatally with hyperammonemia, acidosis, and ketosis. Lifelong management leans on protein-restricted diet plus precursor-free medical food (Mead Johnson Propimex, Nutricia MMA/PA Anamix, Nutricia OA1, MJN XLeu Maxamum for IVA), L-carnitine for organic-acid esterification and renal excretion, glycine for IVA, hydroxocobalamin IM for cobalamin-responsive MMA, biotin for PA variants, and ammonia scavengers (sodium benzoate, sodium phenylbutyrate, Ravicti glycerol phenylbutyrate from Horizon/Amgen FDA 2013, Carbaglu carbamylglutamate from Recordati FDA 2010) during decompensation. Combined liver-kidney transplant is increasingly curative for severe MUT mut0 MMA with nephropathy; heart transplant rescues refractory PA cardiomyopathy. The editorial frontier is Moderna's mRNA-3705 (MMA) and mRNA-3927 (PA) Phase 1/2 programs delivering mRNA encoding the missing enzymes. Patient infrastructure: Organic Acidemia Association (OAA, oaanews.org), PA Foundation, MMA Foundation, Clinic for Special Children Strasburg PA (Amish GA1 cohort), European E-IMD registry. Ninety-seventh deliberate non-elevation of community peptides.
What changes during this transition
Patient infrastructure is Organic Acidemia Association (OAA, oaanews.org), PA Foundation, MMA Foundation, the Glutaric Acidemia Type 1 family network, and the Clinic for Special Children in Strasburg PA — the same Lancaster County center that anchors the Amish MSUD cohort runs a major GA1 program for the Old Order Amish founder population. The European E-IMD registry coordinates cross-border outcome data for intoxication-type IEMs. US newborn screening detects all four on RUSP via C3, C5, and C5DC acylcarnitines. Classical forms present in the neonatal period; adult survivors increasingly live with MMA nephropathy, PA cardiomyopathy, optic atrophy, and post-transplant immunosuppression — a population where peptide questions now arrive. No peptide on this catalog has been characterized in MMA, PA, IVA, or GA1. The disease-modifying interventions are protein restriction with precursor-free medical food, L-carnitine, glycine (IVA), B12 (cobalamin-responsive MMA), biotin (PA variants), ammonia scavengers during crises, and — for selected severe cases — combined liver-kidney or heart transplantation, with Moderna's mRNA-3705 and mRNA-3927 Phase 1/2 programs as the editorial frontier. GH-axis peptides drive propionate and methylmalonate flux from precisely the catabolic pools the diet is engineered to suppress. The same mechanism-vs-protective-diet collision that runs through MSUD (batch 160) runs through the organic acidemias here. Deliberate non-elevation: the editorial honest move is to defer to the metabolic team rather than imply a peptide belongs in this protocol. Ninety-seventh deliberate non-elevation.
Important caveat
No peptide on this catalog has been characterized in MMA, PA, IVA, or GA1. The disease-modifying interventions are protein restriction with precursor-free medical food (Mead Johnson Propimex, Nutricia MMA/PA Anamix, Nutricia OA1, MJN XLeu Maxamum for IVA), L-carnitine, glycine (IVA), B12 (cobalamin-responsive MMA), biotin (PA variants), ammonia scavengers during crises (sodium benzoate, Ravicti FDA 2013, Carbaglu FDA 2010), and — for selected severe cases — combined liver-kidney or heart transplantation, with **Moderna's mRNA-3705 (MMA) and mRNA-3927 (PA) Phase 1/2 programs** as the editorial frontier. **GH-axis peptides drive propionate and methylmalonate flux from precisely the catabolic pools the diet is engineered to suppress**. Deliberate non-elevation: the editorial honest move is to defer to the metabolic team — Clinic for Special Children Strasburg PA for Amish GA1, OAA + PA Foundation + MMA Foundation network, European E-IMD registry — rather than imply a peptide belongs in this protocol. **Tesamorelin Rule 6 SHARPEST** — HIV-LD FDA label does NOT propagate. **Semaglutide**: appetite suppression directly threatens medical-food adherence + catabolic flux risk during titration. **NO Juno library peptide is surfaced as an organic acidemia discovery card — 97th deliberate non-elevation**. WADA: BPC-157 (S0) + CJC-1295 + ipamorelin + tesamorelin (S2) prohibited at all times.
Want this list to grow? The library is editorial — if there’s a peptide you think belongs on this page with documented or mechanistically-clear evidence, send us a note with the citation and we’ll review it under the same evidence-tier discipline as every other entry.