Phosphorylated TDP-43 immunoreactivity correlates with stress granule assembly in neurons and glia, caspase-3 activation, and neurodegeneration (Yan et al., 2025)
Abstract Glucose-6-phosphate dehydrogenase (G6PD) deficiency, one of the most common human genetic enzymopathies, is caused by over 160 different point mutations and contributes to the severity of many acute and chronic diseases associated with oxidative stress, including hemolytic anemia and bilirubin-induced neurological damage particularly in newborns

Download our corporate fact sheet Select Phase(s) Select Therapeutic Area(s) Select Modality(s) Biologics Genetic Medicines Cell Medicines Therapeutic Area(s) Oncology Modality Cell therapy Indication Ovarian cancer Target MUC16 Molecule Name ALN-5288 In collaboration with Alnylam Therapeutic Area(s) Neurology Modality Gene silencing Indication Alzheimer's Disease Target MAPT Therapeutic Area(s) Cardiovascular/Metabolic Modality Gene silencing Indication People with dyslipidemia Target APOC3 Molecule Name ALN-CFB In collaboration with Alnylam Therapeutic Area(s) Hematology, Rare Disease Modality Gene silencing Indication Paroxysmal Nocturnal Hemoglobinuria (PNH) Target CFB Therapeutic Area(s) Cardiovascular/Metabolic Modality Gene silencing Indication Metabolic dysfunction-associated steatohepatitis (MASH) Target CIDEB Molecule Name ALN-F1202 In collaboration with Alnylam Therapeutic Area(s) Hematology Modality Gene silencing Indication Healthy volunteers Target Factor XII Molecule Name ALN-HTT02 In collaboration with Alnylam Therapeutic Area(s) Neurology Modality Gene silencing Indication Huntingtons disease Target HTT Molecule Name ALN-PNP In collaboration with Alnylam Therapeutic Area(s) Cardiovascular/Metabolic Modality Gene silencing Indication Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) Target PNPLA3 Therapeutic Area(s) Neurology Modality Gene silencing Indication Parkinson's Disease Target SNCA Molecule Name ALN-SOD* In collaboration with Alnylam *on U.S

Understanding these timelines is crucial for ensuring medication efficacy and patient safety
Use of acetyl L- carnitine and propionyl L- carnitine, or one of their pharmaceutically acceptable salts, for the preparation of a medicament and/ or dietetic product for the treatment of erectile dysfunction