Rare Disease Experience

Formed in 2001, Engage Health has worked in a number of disease areas – with over 20 + years experience in the orphan and specialty product arenas – the following is a listing of just some of that experience

Ethylene Glycol Poisoning

Methanol Poisoning

Epidermolysis Bullosa

Bone Metastases

Bone Marrow/Stem Cell Transplant

CLL, NHL

Mycosis Fungoides

Neuroblastoma

Prostate Cancer

Angina

Essential Thrombocythemia

Hepatic Porphyria

HoFH

Patent Ductus Arteriosis

Recalcitrant Hypertension

Rhythm disorders

Sickle Cell Anemia

Alzheimer’s Disease

Chronic Intractable Pain

Dystonia

Movement Disorders

Narcolepsy

Parkinson’s Disease

Status Epilepticus

Traumatic Brain Injury

Dravet Syndrome

Cleft palate

Acromegaly    

Amyloidosis

Congenital Sucrase Isomaltase Deficiency

Crohn’s Disease

Cystinosis

Cystinuria

dRTA; inherited and acquired

Esophageal Varices

Fecal Impaction

Gastroparesis

Struvite Stones

Congenital Chronic Cholestasis

NAFLD/ NASH

Hereditary Angiodema

Batten Disease; CLN2, CLN3

Fabry

Farber

Gaucher

Huntington Disease

MPS I, MPS II, MPS III A, MPS III B, MPS VI, MPS VII

Nephropathic Cystinosis

Pompe

Wolman Disease (LAL Deficiency)

Niemann Pick Type C

AADC Deficiency

CPT I, CPT II

Creatine Transporter Deficiency (CTD)

Fatty Acid Oxidation Disorders (VLCAD, LCHADD, CPT2, CACT)

Glucose Transporter Deficiency Type I

Hepatic Encephalopathy

Homocystinuria

Lipodystrophy

Mitochondrial Disorders (LCXHAD, MCAD, SCAD, VLCAD)

NAGs Deficiency

Neurofibromatosis Type 1

Obesity due to MC4R Defects

Urea Cycle Disorders

X-Linked Hypophosphatemic Rickets Hypophosphatasia (HPP)

Achondroplasia

Fibrodysplasia Ossificans Progressiva

Fracture Repair

Hereditary Inclusion Body Myopathy

Hereditary Multiple Exostoses/ Osteochondromas

Occulopharyngeal Muscular Dystrophy

Spinal Fusions

Spinal Muscular Atrophy

A-AION and P-AION

Retinitis Pigmentosa, Lebers Congenital Amourosis

Asthma

COPD

Pulmonary Arterial Hypertension

Breast Density, Breast CA

Diagnostic for Detection of Cervical Cancer

Menopause

Nausea and Vomiting of Pregnancy

Pre and postnatal supplementation