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About 515 results

ALLMedicine™ Methylmalonic Acidemia Center

Research & Reviews  188 results

A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
https://clinicaltrials.gov/ct2/show/NCT04899310

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...

Clinical and Laboratory Study of Methylmalonic Acidemia
https://clinicaltrials.gov/ct2/show/NCT00078078

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 ...

Validation of a targeted metabolomics panel for improved second-tier newborn screening.
https://doi.org/10.1002/jimd.12591
Journal of Inherited Metabolic Disease; Mak J, Peng G et. al.

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...

Newborn screening for inborn errors of metabolism in a northern Chinese population.
https://doi.org/10.1515/jpem-2022-0543
Journal of Pediatric Endocrinology & Metabolism : JPEM; Liu G, Liu X et. al.

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...

A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
https://clinicaltrials.gov/ct2/show/NCT04836494

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.

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Drugs  1 results see all →

Clinicaltrials.gov  14 results

A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
https://clinicaltrials.gov/ct2/show/NCT04899310

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...

Clinical and Laboratory Study of Methylmalonic Acidemia
https://clinicaltrials.gov/ct2/show/NCT00078078

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 ...

A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
https://clinicaltrials.gov/ct2/show/NCT04836494

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.

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
https://clinicaltrials.gov/ct2/show/NCT05040178

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...

Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia
https://clinicaltrials.gov/ct2/show/NCT04581785

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...

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News  3 results

AstraZeneca Pays 660% Premium for Gene Therapy Firm LogicBio
https://www.medscape.com/viewarticle/981755

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...

Table of Surrogate Endpoints That Were the Basis of Drug Approval or Licensure
http://www.fda.gov/drugs/development-resources/table-surrogate-endpoints-were-basis-drug-approval-or-licensure

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...

AstraZeneca pays 660% premium for gene therapy firm LogicBio undefined Gallery Gallery Gallery Gallery Open in new tab Open in new tab Open in new tab Open in new tab Open in new tab Open in new ta...
https://www.reuters.com/business/healthcare-pharmaceuticals/astrazeneca-pays-660-premium-gene-therapy-firm-logicbio-2022-10-03/

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...

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