Founded in 1926, the Seth Gordhandas Sunderdas Medical College (GSMC) and the King Edward Memorial (KEM) Hospital are amongst the foremost teaching and medical care institutions in India. The medical college (school) provides training to about 2000 students in undergraduate, postgraduate and superspeciality medical courses; in undergraduate and postgraduate physical and occupational therapy; Masters and PhD courses in various allied specialties. With about 390 staff physicians and 550 resident doctors, the 1800 bedded hospital treats about 1.8 million out-patients and 85,000 in-patients annually Funded mainly by the Municipal Corporation of Greater Mumbai , these institutions render yeomen service – virtually free of cost – mostly to the underprivileged sections of the society. Experience in Rare diseases: Currently, KEM Hospital in Mumbai is a major referral center for genetic disorders with referrals of 600 to 800 patients every year from all over India. Common and rare genetic diseases (chromosomal disorders, microdeletion/ microduplication syndromes, skeletal dysplasias, inborn errors of metabolism, malformation syndromes, families for prenatal counseling and prenatal diagnosis, premarital counseling, recurrent reproductive losses, disorders of sexual development and many other categories) are evaluated and treated every year at the centre. More than 30 patients with lysosomal storage disorders like Gaucher disease, Pompe disease, mucopolysaccharidosis type II and type VI have received enzyme replacement therapy. Children with spinal muscular atrophy are also receiving treatment with antisense oligonucleotide (ASO) therapy with Nusinersen (Spinraza) since the past two years. Apart from other rare diseases, the institute has done pioneering work on Budd Chiari syndrome, a rare vascular liver disease affecting children and young adults which if recognized and intervened early can prevent worsening of liver functions. Gastrointestinal diseases like “very early onset” inflammatory bowel disease, genetically predisposed pancreatitis, endocrine disorders like neonatal diabetes, neuroendocrine tumours and hematological non- malignant childhood diseases like congenital dyserythropoeticanemia, Diamond Blackfan syndrome, and coagulation factor deficiencies are a few of the wide spectrum of rare diseases that the institute caters to.