Drug development

Question 1

what is spinal muscular atrophy

Answer 1

Rare genetic disease affecting Motor nerve cells in spinal cord and is the leading genetic cause of onfa t mortality

Cused by inherited faulty SMN1 gene

Question 2

what is zolgensma

Answer 2

adeno associate virus vector based genre therapy delivering a function SMN1 gene to to motor neurone cells

Question 3

what is gene therapy

Answer 3

Experimental technique using genes to treat/prevent disease

It can
-replacing mutated gene with healthy copy
-inactivating a mutated gene
-introducing a new gene into the body to fight a disease

Question 4

Give some clinical uses of Glucocorticoide

Answer 4

Skin - eczema, psoriasis

Lung- asthma, COPD

GI- inflammatory bowel disease

Musculoskeletal- inflammatory arthritis

Eye- acute iritis, blepheritis

CNS- multiple sclerosis
Renal- acute vasculitis, anti-rejection for graft

Infectious diseases- improves outcome in meningitis
Haematology- auto-immune thrombocytopaenia

Malignant disease- reduce brain swelling in cerebral metastases

Pregnancy- fetal lung maturation in premature delivery

Adrenal failure- replacement therapy