- Signe Sparre Beck-Nielsen,
- Zulf Mughal,
- Dieter Haffner,
- Ola Nilsson,
- Elena Levtchenko,
- Gema Ariceta,
- Carmen de Lucas Collantes,
- Dirk Schnabel,
- Ravi Jandhyala
- Outi Mäkitie
Read more indepth information on a selection of our research projects.
X-linked hypophosphatemia (XLH) is an inherited disease of phosphate metabolism in which inactivating mutations of the Phosphate Regulating Endopeptidase Homolog, X-Linked (PHEX) gene lead to local and systemic effects including impaired growth, rickets, osteomalacia, bone abnormalities, bone pain, spontaneous dental abscesses, hearing difficulties, enthesopathy, osteoarthritis, and muscular dysfunction. Patients with XLH present with elevated levels of fibroblast growth factor 23 (FGF23), which is thought to mediate many of the aforementioned manifestations of the disease. Elevated FGF23 has also been observed in many other diseases of hypophosphatemia, and a range of animal models have been developed to study these diseases, yet the role of FGF23 in the pathophysiology of XLH is incompletely understood.
The client promoted a medicine for the treatment of type 2 diabetes, one of the key secondary characteristics of which was its beneficial effect on weight.
Healthcare professionals (HCPs) are responsible for the selection of appropriate treatment for their patients.
Pulmonary arterial hypertension (PAH) is a disorder primarily of small pulmonary arteries which results in a progressive rise in pulmonary vascular resistance and right ventricular failure. It consist of 4 classes of increasing severity relating to exercise tolerance.
There were three tyrosine kinase inhibitors approved for the treatment of EGFR+ve non small cell lung cancer.
The community was of the impression that there was no differentiation within this class and that TKI – A had the edge in terms of efficacy, even without any head-to-head data.
Three meta-analyses were conducted, which showed that in terms of efficacy, the three members were indeed equivalent. However, no systematic review and meta-analysis was conducted on the relative safety profiles of the members in the class.
The botulinum toxin (BoNT-A) market comprised three main products.
The aesthetics indication was somewhat limited to the glabellar lines for all. The glabella was one of a possible 10 areas of the face that were currently being treated.
Promotion was limited due to regulatory constraints and there was a commonly held feeling that all three BoNTA’s were equivalent in efficacy and safety.
The rapid onset opioid class was congested with three members, with little clear differentiation between them.
When considering that the BTcP market was still in need of building, this meant that several companies were competing for a relatively small number of patients.
The Breakthrough Cancer Pain market is dwarfed by other pain markets, such as chronic pain.
The concept of BTcP was a relatively new one in that:
It was a specific type of cancer pain that was characterized by high insensity, short onset, short duration pain.