Therapeutic Areas

Endocrine and Metabolic Disease Clinical Development Expertise

Overcoming complexities to bring more metabolic disease treatments to market successfully. 

Metabolic and endocrine diseases are a rising therapy area focus for clinical research due to the high demand for novel therapies that are challenged with proving safety and efficacy. The development process is complicated and full of unknowns, and even the best metabolic therapeutic might not make it to the patients that need it, not because it does not work but because the design, data collection, and analysis were not done right. 

 At Veristat, we have assembled an extraordinary team of scientific-minded experts who have mastered the complexities of running metabolic disease trials. Our team has supported 300+ projects for endocrine/metabolic disorder treatments and the preparation of 30+ marketing applications - 60% of which have been approved or authorized so far! 


Endocrine & Metabolic Projects


in Rare/Ultra-Rare Indications


in Biologic Therapeutics

Specialized Expertise from Consultation to Beyond Submission

Our solutions span the entire clinical development life-cycle - and are offered as a comprehensive, all-inclusive solution or as functional support. We provide strategic guidance for informed decision-making, operational support to mitigate trial risks, the statistical knowledge to prove safety and efficacy, the medical oversight to ensure patient safety, and the regulatory expertise to achieve approval success.

I would like to thank the entire team at Veristat, who worked day and night to submit our DSUR and our IND within thirty days of starting to work with us!

In addition to managing the process, the team worked seamlessly with us in the United States and our partner in Korea. Your team advised and guided us to produce quality submissions to meet our challenging corporate goal of submitting both on time. Due to these extraordinary efforts, we plan to work with you on our second IND submission.
Clinical Stage Biotechnology Company

Endocrine & Metabolic Highlights

Common Indications
Anorexia Hypertriglyceridemia
Diabetes Menopause
Dyslipidemia Molybdenum cofactor deficiency (MoCD)
Gout Nonalcoholic Steatohepatitis (NASH)
Growth Hormone Deficiencies/Abnormalities Obesity
Homozygous Familial Hypercholesterolaemia Osteoporosis
Hypercholesterolemia Polycystic Ovary Syndrome (PCOS)
Hyperoxaluria Vasomotor Symptoms (VMS)
Rare Indications
Achondropasia Lipodystrophy
Adrenoleukodystrophy (ALD) Lipoprotein Lipase (LPL) Deficiency
Alagille Syndrome ALGS (liver disease) Lysosomal Acid Lipase (LAL) Deficiency
Alpha-mannosidosis (AM) Maroteaux-Lamy Syndrome
Bardet-Biedl Syndrome (BBS) McArdle Disease
Cerebral Adrenoleukodystrophy (CALD) Metachromatic Leukodystrophy (MLD)
Cerebrotendinous Xanthomatosis (CTX) Methylmalonic Acidemia
Congenital Adrenal Hyperplasia (CAH) Molybdenum Cofactor Deficiency (MoCD)
Duchenne Muscular Dystrophy (DMD) Mucopolysaccharidosis IIIB & VI
Ehlers-Danlos Syndrome (EDS) Mutase-Deficient Methylmalonic Acidemia (MMA)
Fabry Disease Myotonic Dystrophy
Familial Amyloid Polyneuropathy Neuroendocrine Tumors (NET)
Familial Chylomicronemia Syndrome (FCS) Pediatric Congenital Athymia
Fatty Acid Oxidation Disorder (FAOD) Phenylketonuria (PKU)
Fibrodysplasia Ossificans Progressvia (FOP) Pompe Disease
Galactosemia Primary Mitochondrial Myopathy (PMM)
Gaucher Disease Progressive Familial Intrahepatic Cholestasis (PFIC)
Hereditary Transthyretin (hATTR) Amyloidosis Pyruvate Kinase Deficiency
Homocystinuria Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Homozygous Familial Hypercholesterolaemia (HoFH) Sanfilippo Syndrome
Hypoleptinemic Dysmetabolic Disorder Wilson's Disease
Hypoparathyroidism X-Linked Hypophosphatemia
Limb-girdle Muscular Dystrophy (LGMD) X-linked Myotubular Myopathy

Case Study_First Gene Therapy Approved in Europe_Card Module


From Phase I rescue to long-term follow up, our team’s expertise and dedication helped the client cross the finish line with the first gene therapy ever to be approved in Europe.

Find out how our clinical research expertise can help with your next study or program.


Learn More with These Resources

Case Study

Full-Service, Randomized Pancreatic Cancer Phase II Trial

Webinar Presentation

Future Trends on European Regulations on Medicines

Fact Sheet

Endocrine and Metabolic Fact Sheet