Cystic Fibrosis Treatments
The Pulmonary Clinical Practice Guidelines Committee of the Cystic Fibrosis (CF) Foundation provided updated recommendations on the use of available chronic treatments for Cystic Fibrosis based on current evidence.
Metabolic Disorders
Continuing Semaglutide May Aid Weight Loss in Overweight, Obese
Maintaining treatment resulted in continued weight loss during additional 48 weeks compared with switching to placebo
Risk for Severe Outcomes in COVID-19 Increases With Increasing BMI
Risks for hospitalization, intensive care unit admission, and death increased sharply with higher BMIs
Semaglutide May Aid Weight Loss in Obese Patients With T2DM
Estimated change in mean body weight to week 68 was −9.6 percent with semaglutide 2.4 mg versus −3.4 percent with placebo
Tesomet Granted Orphan Drug Status for Prader-Willi Syndrome
Tesomet is an investigational fixed-dose combination therapy of tesofensine, a triple monoamine reuptake inhibitor, and metoprolol, a beta-1 selective blocker.
Nulibry Approved for Molybdenum Cofactor Deficiency Type A
Nulibry replaces missing cyclic pyranopterin monophosphate, which is needed to reduce levels of neurotoxic sulfites.
Early Treatment With Galsulfase Appears to Slow Disease Progression in MPS VI
The aim of this multicenter, prospective analysis was to determine the long-term outcomes of treatment of MPS VI patients with galsulfase beginning in infancy.
Case Series Describes Hydroxychloroquine Use, Toxicity in Fabry Disease
A true diagnosis of Fabry disease may be delayed or the condition may be completely misdiagnosed, as it often masquerades as a rheumatological disease.
Clinical Benefits Observed With Investigational Gene Therapy for ES-EJ Metachromatic Leukodystrophy
Currently, there are no approved treatments for MLD, a lysosomal storage disease caused by a deficiency in arylsulfatase A.
Does Gaucher Disease Up SARS-CoV-2 Infection Risk?
Patients with Gaucher disease do not appear to be at increased risk for SARS-CoV-2 infection, according to findings presented at the 17th Annual WORLDSymposium.
