Pfizer Ends Development of Drug for LMNA-Related Dilated Cardiomyopathy
PF-07265803 is an investigational selective, oral, small-molecule inhibitor of the p38a mitogen activated protein kinase pathway.
Inborn Errors Of Metabolism
Patisiran Promising for Transthyretin-Mediated Amyloidosis With Cardiomyopathy
The APOLLO-B study included patients 18 years of age and older with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy.
Eplontersen Bests Placebo in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Study
Eplontersen is an investigational ligand-conjugated antisense medicine designed to reduce the production of TTR protein to treat both hereditary and nonhereditary forms of ATTR.
Imcivree Approved for Weight Management in Bardet-Biedl Syndrome
Imcivree is a melanocortin 4 receptor (MC4R) agonist that re-establishes MC4 receptor pathway activity to reduce hunger and promote weight loss.
MPR Weekly Dose Podcast #120
FDA panel vote on COVID-19 vaccines for young children; new drug approved to treat alopecia; Paxlovid tested in standard risk individuals.
Amvuttra Approved for Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis
Amvuttra is a subcutaneously-administered RNAi therapeutic designed to block the production of wild-type and mutant transthyretin protein.
FDA Panel Votes in Favor of Gene Therapy for Cerebral Adrenoleukodystrophy
Eli-cel is an investigational one-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells.
ALK-2 Inhibitor Fast Tracked for Fibrodysplasia Ossificans Progressiva
BCX9250 is an investigational activin receptor-like kinase-2 (ALK-2) inhibitor designed to stimulate normal bone growth and renewal.
Omaveloxolone Gets Priority Review for Friedreich Ataxia
Omaveloxolone is an investigational Nrf2 activator.
