familial hypercholesterolemia
Jun 10, 2025
Amylyx’s AMX0114 Fast Tracked by FDA for ALS; Cellectar’s Iopofosine I 131 Granted FDA Breakthrough in Waldenstrom Macroglobulinemia; YolTech’s YOLT-101 Clears FDA IND for Familial Hypercholesterolemia; Oncovita’s MVdeltaC Gets FDA Orphan Tag for Pleural Mesothelioma; Merck’s ENFLONSIA Approved for RSV Prevention in Infants
Amylyx Pharmaceuticals Receives FDA Fast Track Designation for AMX0114 in ALS Amylyx Pharmaceuticals announced that the FDA has granted Fast Track designation to its investigational antisense oligonucleotide (ASO), AMX0114, for the treatment of amyotrophic lateral sclerosis (ALS). The candidate targets calpain-2...
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Oct 10, 2022
PCSK9 Inhibitors: A New Era for Cholesterol Management
Proprotein convertase subtilisin/Kexin type 9 (PCSK9) affects LDL receptor degradation and decreases the clearance of circulating LDL particles, which is crucial in cholesterol metabolism. PCSK9 is predominantly generated by hepatocytes, with additional sources including the intestines and kidneys. PCSK9 reduces LD...
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