Rare diseases and rare drugs

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1. “Spinal Muscular Atrophy (SMA)” is a rare genetic disease.
2. In India, there are families whose children are suffering from this progressive disease.
3. Due to SMA, children cannot walk, and, the nightmare does not end here as it is a progressive degenerative disease which means the kids may potentially see deterioration in function of their other limbs – hands, spines and even lungs.
4. Limited data suggest that survival has increased in patients with SMA type 1 born from 1995 through 2006 compared with those born from 1980 to 1994
5. Ventilation for >16 hours a day, use of mechanical insufflation-exsufflation device, and gastrostomy tube feeding were significantly and independently associated with prolonged survival, while year of birth was not. Thus, longer survival in the later time period appears to be related to more aggressive care.
6. Treatments that enhance the level of SMN protein may be available in the future
7. Gene therapy using an adeno-associated virus vector to augment spinal cord SMN expression has shown promise in a mouse model of SMA
8. Another promising approach involves intracerebroventricular or systemic injection of antisense oligonucleotides that effectively restore SMN expression
9. Drugs that selectively modify the splicing of the survival motor neuron gene 2 (SMN2) messenger RNA also have a potential therapeutic role
10. There is a FDA approved drug viz. “Nusinersen” under development
11. The Drug has shown promising results after under going clinical trials.
12. Bogen (the company which has developed this drug)
13. The drug is not yet launched in India
14. Estimated cost >$100,000 per year
15. No Health Insurance Company in India covers such kind of disease or costs

Once introduced how to make such drugs affordable in India.

Should CRS, insurance companies and government subsidy under one roof be the answer for such diseases and drugs?

Give anti-hypertensive drugs at night

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Anti-hypertensive drugs should be taken at night.

Uncontrolled blood pressure can lead to heart attacks, paralysis and heart failure. Most such attacks occur in the early morning hours. Pulse, blood pressure and thickening of platelets are all higher in the early morning hours.

Controlling early morning blood pressure can reduce cardiovascular mortality.

Among patients with chronic kidney disease and high blood pressure, taking at least one antihypertensive drug at bedtime significantly improves blood pressure control, with an associated decrease in risk for cardiovascular events, according to a study published in the Journal of the American Society of Nephrology.

The study included 661 patients with chronic kidney disease who were randomly assigned either to take all prescribed anti BP drugs on awakening or to take at least one of them at bedtime. Patients were followed for a median of 5.4 years; during that time, patients who took at least one BP–lowering drug at bedtime had approximately one third of the cardiac risk compared with those who took all medications on awakening.

A similar significant reduction in cardiac deaths, heart attacks and paralysis was noted with bedtime dosing. Patients taking their medications at bedtime also had a significantly lower mean BP while sleeping.

For each 5 mmHg decrease in mean sleep–time systolic upper BP, there was a 14% reduction in the risk for cardiovascular events during follow–up.

Potential explanation for the benefit of night time treatment may be associated with the effect of night time treatment on urinary albumin excretion levels. Urinary albumin excretion is significantly reduced after bedtime, but not morning, treatment.

Sodium in drugs can be dangerous

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Using effervescent, dispersible or soluble drugs on a regular basis leads to greater risk for heart attacks, strokes, and other cardiovascular events.
Regular use of prescribed effervescent and other sodium–containing drugs have a 16% greater risk for nonfatal stroke, nonfatal myocardial infarction (MI), and vascular death (P<0.01), compared with regular users of low or no–sodium versions of the same drug as per Dr Jacob George, at the University of Dundee in Scotland who write in BMJ.

Taking the maximum daily dose of drugs like effervescent aspirin or acetaminophen may exceed the recommended daily limit of sodium. Effervescent paracetamol 500 mg can contain 18.6 mmol of sodium in each tablet.

Sodium-loaded effervescent, soluble or dispersible tablets should be avoided in patients at risk of hypertension.

Current US guidelines recommend that people at low risk for CVD events limit their sodium intake to no more than 2,300 mg (1 teaspoon or 100 mmol/L) per day.

Certain populations, including people over 50, African Americans, diabetics and people with high blood pressure or chronic kidney disease, should limit their daily sodium intake to 1,500 mg.

The American Heart Association (AHA) recommends intake of less than 1,500 mg of sodium a day for everyone.

The World Health Organization (WHO) recommendations call for limiting daily sodium intake to no more than 2,000 mg per day.

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