Methylmalonic Acidemia (MMA) Archives

LogicBio cleared to continue dosing in pediatric MMA trial

Acid Disorders, Clinical Hold, Clinical Trials, FDA, Gene Mutations, Methylmalonic Acidemia (MMA), Pediatric Rare Diseases, R&D, Rare Diseases, Severe adverse event (SAE)

LogicBio Therapeutics was granted the green light to continue dosing participants in the company’s Phase I/II Sunrise trial investigating the safety of LB-001 in patients with methylmalonic acidemia (MMA), characterized by methylmalonyl-CoA mutase (MMUT) gene mutations.

May 9, 2022/by BioSpace

LogicBio Crashes Following Clinical Hold of Pediatric Gene Therapy

Acid Disorders, Adeno-Associated Virus (AAV), Business, Clinical Hold, Clinical Trials, Dosing, FDA/Regulatory, Gene Therapy, Methylmalonic Acidemia (MMA), Pediatric, R&D, Shares

Shares of LogicBio Therapeutics fell more than 57 percent on February 2 after the company announced that its gene therapy for pediatric patients with methylmalonic acidemia was placed on clinical hold by the U.S. Food and Drug Administration.

February 2, 2022/by BioSpace

FDA Awards Fast Track Designation to Multiple Non-Covid-19 Candidates

Acid Disorders, Acute repetitive seizures, Coronavirus Disease (COVID-19) Pandemic, Fast Track Designation, FDA, Gene Therapy, Hypogammaglobulinemia, Imaging Agents, Infections and Myelokathexis) syndrome, Methylmalonic Acidemia (MMA), MPS Type VI, Non-Small Cell Lung Cancer (NSCLC), Phenylketonuria (PKU), Primary Mitochondrial Myopathy (PMM), Solid Tumors, WHIM (Warts

While the world has largely been focused on the development of vaccines and therapeutics for Covid-19, the U.S. Food and Drug Administration has remained busy lining up potential approvals of medications for other diseases and illnesses.

November 4, 2020/by Andrew Humphreys

FDA Slaps Clinical Hold on LogicBio’s Gene Therapy Treatment

Acid Disorders, Clinical Hold, FDA, FDA, FDA/Regulatory, Gene Therapy, Methylmalonic Acidemia (MMA)

Shares of LogicBio plunged more than 25 percent after the company disclosed that the U.S. Food and Drug Administration placed a clinical hold on the proposed gene therapy treatment for methylmalonic acidemia (MMA).

February 11, 2020/by BioSpace