Track record and expertise

We have successfully managed more than 400 projects in the Orphan Drug niche, of which approx. 40% were regulatory & clinical consultancy services (e.g. orphan drug designation, protocol assistance & paediatric investigation plan) and 60% involved actual execution of clinical studies.

Therapeutic Area

Of these projects described above, over 200 involved an orphan disease. Shown below is our orphan experience distributed across our various services, and further split into therapeutic areas.

 

 

Indication

Metabolic Disorders
  • Mucopolysaccharidosis
  • Gaucher’s disease
  • Alkaptonuria
  • Hereditary Tyrosinemia Type 1
  • Cystinosis
  • Mitochondrial oxidative phosphorylation diseases
  • Wilson disease
  • mitochondrial OXPHOS disease
  • MELAS
  • Wilson disease
  • Fabry Disease
  • Pompe Disease
  • Urea Cycle Disorders
  • AL Amyloidosis
  • Cushing Syndrome
  • Fatty Acid Oxidation Disorder
  • Growth Hormone Deficiency
  • Diabetes; Gastroesophageal Reflux; Heartburn; Hypertriglyceridemia; Ulcerative Colitis
  • Cystic Fibrosis
  • Phenylketonuria (PKU)
  • Diabetic Neuropathy; Hyperkalemia; Hypertension
  • Chronic Kidney Disease; Hyperkalemia
  • Diabetic Neuropathy; Hyperkalemia; Hypertension
  • Niemann-Pick
  • HIV-associated lipodystrophy syndrome
  • Iron overload (hereditary haemochromatosis (HHC))
Oncology
  • Renal cell carcinoma
  • Pancreatic cancer
  • Acute Myeloid Leukemia
  • Neuroblastoma
  • AML,NHL,MM
  • Renal cell carcinoma
  • Chronic Lymphocytic Leukemia (CLL)
  • Chronic Myeloid Leukemia (CML)
  • Hepatocellular Carcinoma
  • Glioblastoma Multiforme
  • Cutaneous T-cell Lymphoma
  • Malignant pleural mesothelioma (MPM); nonsquamous nonsmall cell lung carcinoma
  • Anaplastic Astrocytoma; Recurrent Glioblastoma
  • Acute Myeloid Leukemia; Solid Tumor
  • Anaplastic Astrocytoma; Recurrent Glioblastoma
  • Chronic myelomonocytic leukemia (CMML)
  • Chronic Lymphocytic Leukemia
  • Hepatocellular Carcinoma
  • Acute limphoblastic leukaemia
  • Melanoma
  • Mesothelioma
  • Acute limphoblastic leukaemia
  • Medullary Thyroid cancer
  • Cutaneous T-cell lymphoma
  • Acute limphoblastic leukaemia
  • Pancreatic cancer
  • Multiple myeloma
  • Renal cell carcinoma
  • Acute Myeloid Leukemia
  • Cutaneous T-cell lymphoma
Central Nervous System & Neurology
  • Relapsing Remitting Multiple Sclerosis
  • Spinal Muscular Atrophy
  • Coma
  • Relapsing Remitting Multiple Sclerosis
  • Prader Willi Syndrome
  • Amyotrofe laterale sclerosis (ALS)
  • Perinatal Hypoxic Ischemic Enchaphalopathy
  • Transthyretin Amyloidosis
  • Duchenne Muscular Dystrophy
  • Hereditary Inclusion Body Myopathy
  • Restless Leg Syndrome
  • Peripheral Neuropathy
  • Cervical Dystonia
  • IGF Deficiency
  • Amyloidosis ? Dutch Type; Hereditary Cerebral Hemorrhage
  • Muscular Dystrophy
  • Hereditary Inclusion Body Myopathy
  • X-Linked Myotubular Myopathy
  • Fragile x syndrome
  • Rett syndrome
  • Friedrich’s ataxia
  • Spinal Muscular Atrophy
  • Congenital Myasthenic syndrome
  • Spinal Muscular Atrophy
Immunology & Infectious Disease
  • Scleroderma
  • Heriditary Angioedema
  • GvH disease
  • Dengue Fever
  • Eosinophilic esophagitis
  • Systemic sclerosis
  • chemotherapy-induced neutropenia (CIN)
  • Castleman’s Disease
  • Lupus
  • Candidiasis
  • Adrenocortical insufficiency
  • HIV-associated lipodystrophy syndrome
Opthalmology
  • Acanthamoeba Keratitis
  • chronic non-infectious Uveitis
  • Fuch’s dystrophy
  • Acute Optic Neuritis
  • Acute Non-Arthretic Ischemic Neuropathy
  • Macular Degeneration
    Diabetic Retinopathy
  • Keratoconus
  • Choroidal Neovascularization; Macular Degeneration
  • Retinal Vein Occlusion; Uveitis
  • Geographic Atrophy; Macular Degeneration
  • Choroidal Neovascularization; Macular Degeneration
  • Macular Degeneration
  • Achromatopsia
  • Juvenile retinoschisis
  • Retinitis pigmentosa
  • Nystagmus
Cardiovascular & haematology
  • Hereditary antithrombin III deficiency
  • Buerger’s disease
  • Calciphylaxis
  • pulmonary arterial hypertension
  • Thalassemia
  • Hypertriglyceridemia
  • Myelodysplastic Syndrome
  • Thalassemia
  • Raynaud Phenomenon; Systemic Sclerosis
  • Calciphylaxis; Uremic Calciphylaxis
  • Acute Myeloid Leukemia; Myelodysplastic Syndrome (MDS)
  • Polycythemia Vera
  • Hematologic Malignancies; Myelodysplastic Syndrome (MDS)
  • Congenital factor X deficiency
  • Pulmonary Hypertension
Pulmonology
  • Idiopathic pulmonary fibrosis
  • Bronchopulmonary Dysplasia
  • congenital diaphragmatic hernia
  • Acute Respiratory Distress Syndrome
  • Idiopathic Pulmonary Fibrosis
  • Severe pneumonia
  • Cystic Fibrosis
  • Severe pneumonia
Nephrology
  • ADPKD
  • Primary hyperoxaluria
  • Alport Syndrome
  • Primary hyperoxaluria
  • Primary Biliary Cirrhosis
  • Alport Syndrome
  • Cushing’s Syndrome
  • Nephrotic syndrome
  • Creatine transporter defect
  • Nephrotic syndrome
Dermatology
  • Burns
  • Netherton Syndrome (EMA)
  • Netherton Syndrome (FDA)
  • Psoriasis
Gastroenterology
  • Barrett Esophagus
  • Gastroparesis

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.

Planetenweg 5
2132 HN Hoofddorp
The Netherlands
Tel: +31-23-55 63 220
Fax: +31-23-55 63 230
Email: info@psr-group.com
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