Casus 2 Myasthenia Gravis

Question 1

What muscles are weak in MG?

Answer 1

• eyes
• swallowing
• breathing muscles
• limbs
  onset in the evening of the day

Question 2

How is MG diagnosed

Answer 2

1. Antibody test: anti Ach antibodies or MuSK antibodies.
2. Electrophysiology: when stimulated repetitively, the compound neuron activation drops due to the lack of ACHR

Question 3

How is MG treated?
long and short acting

Answer 3

Long acting: Corticosteroids or thymectomy in case of a thymoma.
Short acting: intra-venous immune globulin can be given in a myasthenic crisis.

Question 4

How many people suffer MG the disease on 100.000 people?

Answer 4

12

Question 5

Which two groups are at risk of developing MG?

Answer 5

Women at the age of 40 and men at the age over 60

Question 6

which two types of trials are there?

Answer 6

interventional and observational

Question 7

which type of interventional trial are there?

Answer 7

• Prevention trials: trials where the participants do not have the disease.
• screening trials: this can help for people who are in the first stages of the disease.
• Treatment trials: often random so an RCT with two groups, one receiving the medicine and the other placebo.

Question 8

what type of observational trials are there?

Answer 8

• Cohort study: following a group of people over a certain period of time. Afterward, research why a certain group received the disease. can be prospective or retrospective.
• Case control: in this case there is a case group(patient) and a control(healthy) group that are compared to find out why the patients developed the disease.
• Cross- sectional: this aim is to find out who was exposed to certain risk factors and is fast cheap but unreliable.

Question 9

what are the four stages of drug development?

Answer 9

0: research
1: testing on healthy subjects for side-effects.
2: testing on patients with the intended to cure disease.
3: more testing on multiple groups to improve safety in the long term.
4: once FDA approved further investigation on the certain drug.

Question 10

What requirements are needed before you can start pre-clinical testing?

Answer 10

• there needs to be tested on two types of animal, one non rodents like dogs/monkeys
• there must be a summary of in vivo and in vitro research results, including previous pre-clinical testing.
• the drug should be reasonably behaved the same in humans than in the testing animals.

Question 11

How does a proteasome inhibitor work?

Answer 11

It reduces plasma cells and total serum IgG and autoantibodies

Question 12

Where in the body is MG effecting physiology?

Answer 12

In the neuromuscular junction

Question 13

What types of antibodies activate the complement and MAC in MG?

Answer 13

IgG1 and IgG3

Question 14

What is seen on an EMG in patients suffering MG?

Answer 14

Loss of compound motor action potential, leading to lower muscle fibre stimulation.