Data presented by Mirum Pharmaceuticals at the virtual 2021 International Liver Congress demonstrated the safety and tolerability of maralixibat during 5 years of treatment in patients with Alagille syndrome (ALGS). Mirum also presented results of a multinational survey of patients with intrahepatic cholestasis of pregnancy (ICP), which showed the considerable burden of ICP and emphasized…
Removing a single gene engaged in embryonic liver development eliminated the bile ducts in newborn mice, researchers found in a study recently published in Cell Reports. Despite this significant flaw in their bile excretion system, the small and yellow-colored mice survived for up to 8 months and were physically active. The data suggest why certain…
Research published in Science Advances found that safe and targeted delivery of nanoparticle (NP)-based anti-inflammatory cytokines initiates a specific T-cell response, thereby strengthening the muscles affected by Duchenne muscular dystrophy (DMD). Inflammation plays a crucial role in disease progression in muscular dystrophies, a group of chromosomal disorders which lead to progressive loss of muscle mass…
Platelet-derived growth factor-AA (PDGF-AA) could function as a biomarker to track the progression of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) according to the results of a study published in Frontiers of Neurology. DMD and BMD are X-linked recessive disorders caused by mutations of the DMD gene. Both DMD and BMD are slow-developing…
Research published in the Asian Journal of Surgery showed that high-frequency ultrasound and the Thyroid Imaging Reporting and Data System (TI-RADS™) effectively evaluated patients with medullary thyroid carcinoma (MTC). MTC is more malignant than other segregated thyroid cancers because it is highly aggressive, has a poor diagnosis, and is unresponsive to conventional chemotherapy or radiotherapy…
Presymptomatic infants with spinal muscular atrophy (SMA) who were treated with risdiplam (Evrysdi™) reached significant motor milestones such as sitting unaided, rolling, crawling, standing, and walking independently, according to preliminary data presented at the virtual 2021 Spinal Muscular Atrophy Conference. The data come from RAINBOWFISH, an ongoing open-label, single-arm, multicenter study investigating the efficacy, safety,…
Research published in the Annals of the American Thoracic Society found that a low dose of azithromycin was ineffective in controlling chronic cough in patients with idiopathic pulmonary fibrosis (IPF). Patients with IPF frequently suffer from recurring chronic coughs, which affect their standard of living. Studies have shown that azithromycin alleviates the pain caused by…
The US Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for inhaled treprostinil (Tyvaso DPITM) and granted it a priority review for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). Mannkind Corporation and United Therapeutics described the announcement as a significant milestone. Michael Castagna,…
The US Food and Drug Administration (FDA) has approved orphan drug designation for PT001, an investigational treatment from PulmoSim for pulmonary arterial hypertension (PAH) which targets several responsible pathways in an aim to provide a more effective treatment. Orphan drug designation is used by the FDA’s Office of Orphan Products Development (OOPD) to support the…
