Track record and expertise

We have successfully managed more than 300 projects of which approx. 20% were regulatory & clinical consultancy services (e.g. orphan drug designation, protocol assistance & paediatric investigation plan) and 80% involved actual execution of clinical studies.

Therapeutic Area

Of these projects described above, over 120 involved an orphan disease. Shown below is our orphan experience distributed across our various services, and further split into therapeutic areas.

Indication

Oncology
  • Acute Myeloid Leukemia
  • AML,NHL,MM
  • CML
  • Cutaneous T-cell lymphoma
  • Head & Neck Carcinoma
  • Neuroblastoma
  • Non-Hodgkin’s Lymphoma
  • Pancreatic cancer
  • Renal cell carcinoma
  • Syringocystadenoma papilliferum
Central Nervous System & Neurology
  • ALS
  • Amyotrofe laterale sclerosis
  • Cerebral Ataxia with Hypomyelination
  • Coma
  • Congenital Myasthenic syndrome
  • Duchenne Muscular Dystrophy
  • Friedrich’s ataxia
  • Perinatal Hypoxic Ischemic Enchaphalopathy
  • Prader Willi Syndrome
  • Relapsing Remitting Multiple Sclerosis
  • Spinal Muscular Atrophy
  • Transthyretin Amyloidosis
Cardiovascular & haematology
  • IgM Concentrate
  • Pulmonary arterial hypertension
  • Buerger’s disease
  • Calciphylaxis
  • IgM Concentrate
  • Abdominal Aortic Anerusm
  • Haemophilia
  • Haemophilia A
  • Hereditary antithrombin III deficiency
  • Idiopathic Thrombocytopenic Purpura (ITP)
  • Paroxyzmal noctural hemoglobinuria (PNH)
  • Pulmonary Hypertension
  • Sickle Cell Disease
  • Thalassemia
Metabolic Disorders
  • Alkaptonuria

  • Cystinosis

  • Gaucher’s disease

  • GH deficiency

  • Hereditary Tyrosinemia Type 1

  • MELAS

  • Mercury Toxicity

  • Mitochondrial oxidative phosphorylation diseases

  • Mitochondrial OXPHOS disease

  • Mucopolysaccharidosis

  • Wilson disease

Nephrology
  • ADPKD
  • Alport Syndrome
  • Creatine transporter defect
  • Nephrotic syndrome
  • Primary Hypeoxraluria
Immunology & Infectious Disease
  • Chemotherapy-induced neutropenia (CIN)
  • Cytomegaloviral infections
  • Dengue Fever
  • Eosinophilic esophagitis
  • GvH disease
  • Heriditary Angioedema
  • Scleroderma
  • Systemic sclerosis
Pulmonology
  • Bronchopulmonary Dysplasia
  • Congenital diaphragmatic hernia
  • Cystic Fibrosis
  • Idiopathic pulmonary fibrosis
Opthalmology
  • Acanthamoeba Keratitis
  • Acute Optic Neuritis
  • Chronic non-infectious Uveitis
  • Fuch’s dystrophy
Dermatology
  • Burns
  • Netherton Syndrome
Gastroenterology
  • Bile Acid Diarrhea
  • Short Bowel Syndrome
Endocrinology
  • Cystinuria

Partners

Through and with our partners PSR offers a range of services all focussed on the Rare disease niche. Together we will help you define the right approach, develop a clear strategy and implement an optimal solution that not only increases operational efficiency and reduces risks, but also facilitates the optimal outcome of a project.

Antareslaan 41
2132 JE Hoofddorp
The Netherlands
Tel: +31 23 303 69 00
Fax: +31 23 303 69 99
Email: info@psr-group.com
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