ALLMedicine™ Methylmalonic Acidemia Center

Jan 30th, 2023 - This study comprises 2 stages; a Dose Optimization Stage followed by an optional Dose Expansion Stage. The study is designed to evaluate multiple doses and dosing intervals of mRNA-3705. In both stages, after confirmation of eligibility, participa...

Jan 26th, 2023 - Methylmalonic acidemia (MMA), one of the most common inborn errors of organic acid metabolism, is heterogeneous in etiology and clinical manifestations. Affected patients with cblA, cblB and mut classes of MMA are medically fragile and can suffer ...

Jan 22nd, 2023 - Improved second-tier assays are needed to reduce the number of false positives in newborn screening (NBS) for inherited metabolic disorders including those on the Recommended Uniform Screening Panel (RUSP). We developed an expanded metabolite pane...

Jan 21st, 2023 - Newborn screening (NBS) for inborn errors of metabolism (IEMs) has been successfully implemented in China. However, the data on the IEM profiles in many regions are lacking. This study aimed to report the incidence, disease spectrum, and genetic p...

Jan 13th, 2023 - This is the first-in-human study with BBP-671 and is designed to provide healthy subjects single- and multiple-dose and patient multidose safety, tolerability, PK, and PD data regarding BBP-671 for future clinical studies.

Jan 30th, 2023 - This study comprises 2 stages; a Dose Optimization Stage followed by an optional Dose Expansion Stage. The study is designed to evaluate multiple doses and dosing intervals of mRNA-3705. In both stages, after confirmation of eligibility, participa...

Jan 26th, 2023 - Methylmalonic acidemia (MMA), one of the most common inborn errors of organic acid metabolism, is heterogeneous in etiology and clinical manifestations. Affected patients with cblA, cblB and mut classes of MMA are medically fragile and can suffer ...

Jan 13th, 2023 - This is the first-in-human study with BBP-671 and is designed to provide healthy subjects single- and multiple-dose and patient multidose safety, tolerability, PK, and PD data regarding BBP-671 for future clinical studies.

Dec 22nd, 2022 - This study is being conducted to obtain short-term and long-term clinical safety information from adult and pediatric patients treated for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA). This is an observational/non...

Dec 8th, 2022 - The SUNRISE trial is a first-in-human (FIH), open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and preliminary efficacy of a single intravenous infusion of hLB-001 in pediatric patients with MMA characterized by meth...

Oct 4th, 2022 - (Reuters) - Shares of LogicBio Therapeutics skyrocketed on Monday after Britain's AstraZeneca's said it would buy the U.S.-based gene therapy developer at a rare 660% premium for $68 million. LoigBio's shares, which have traded below $1 since Febr...

What is the purpose of the Surrogate Endpoint Table? FDA’s surrogate endpoint table provides valuable information for drug developers on endpoints that may be considered and discussed with FDA for individual development programs. This table also f...

Oct 3 (Reuters) - Shares of LogicBio Therapeutics (LOGC.O) skyrocketed on Monday after Britain's AstraZeneca's (AZN.L) said it would buy the U.S.-based gene therapy developer at a rare 660% premium for $68 million. LoigBio's shares, which have tra...