Added 17 days ago Drug news

NICE guidance rejects the use of Respreeza for alpha-1 antitrypsin deficiency.- CSL Behring.

The National Institute for Health and Care Excellence (NICE) has published draft guidelines that does not support the use by...

Added 1 month ago Drug news

Amicus Therapeutics provides regulatory update on AT GAA for Pompe disease.

Amicus Therapeutics announced regulatory and clinical advancements in its development program AT GAA for Pompe disease. During the third quarter,...

Added 1 month ago Drug news

NICE now recommends Crysvita to treat X-linked hypophosphatemia.- Kyowa Hakko Kirin + Ultragenyx.

The National Institute for Health and Care Excellence (NICE) has now recommended Crysvita (burosumab) from Kyowa Hakko Kirin within its...

UK Commissioning guide: Massive Weight Loss Body Contouring

This document is about guidance for commissioning reconstructive procedures, post massive weight loss and is based on the best available evidence.

Added 4 months ago

Emergency management of acute hypercalcaemia in adult patients

Abnormalities of parathyroid function, bone resorption, renal calcium reabsorption or dihydroxylation of vitamin D may cause regulatory mechanisms to fail and serum calcium may rise.

Added 5 years ago

Acute Hypocalcaemia - for use in adult patients

Acute hypocalcaemia can be life threatening, necessitating urgent treatment. In severe cases, intravenous calcium forms the mainstay of initial therapy, but it is essential to ascertain the underlying cause and commence specific therapy...

Added 5 years ago

A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease.

Drug development for ultra-rare diseases is challenging because small sample sizes and heterogeneous study populations hamper the ability of randomized, placebo-controlled trials with a single primary endpoint to demonstrate valid treatment effects.

Added 1 month ago

Efficacy of High-Dose Liraglutide as an Adjunct for Weight Loss in Patients with Prior Bariatric Surgery.

We sought to determine the efficacy of high-dose liraglutide (3.0 mg once daily) in patients with prior bariatric surgery.

Added 2 months ago

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.

Background: Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin.

Added 3 months ago

CARAT: Canagliflozin vs. placebo for post bariatric patients with persistent type 2 diabetes

This is a prospective, randomized clinical trial for patients with recurrent type 2 diabetes post-gastric bypass surgery that will compare a 6 month course of canagliflozin monotherapy vs. placebo on clinical outcomes of type 2 diabetes.

Added 2 years ago

Setmelanotide for the treatment of early-onset POMC deficiency obesity

The purpose of the study is to determine the effect of setmelanotide (RM-493) on weight and other factors in patients with pro-opiomelanocortin (POMC) deficiency obesity due to rare bi-allelic loss-of function POMC or PCSK1 genetic mutations.

Added 2 years ago

Division-less gastric bypass, step to decrease cost and early nightmare of bariateric surgery

The purpose of this study is to determine the efficacy of of gastric bypass without gastric division as a treatment for morbid obesity.

Added 2 years ago