Ataluren for Cystic Fibrosis

Genetic disorders   have always remained incurable despite the many efforts of medical researchers in the field who are trying to come up with possible cures. Cystic fibrosis is one of such   genetic disorders. It’s usually inherited from parents who are carriers of the faulty gene involved.  Most children living with the disease inherited it from their parents.   Such children may grow up to become adults one day as they still live with the ugly disease.

Over the years, several attempts have been made in curing cystic fibrosis.  Ataluren invention is one of such attempts. It’s a tiny molecular agents designed to make ribosome to be less sensitive to premature stop codons.  The molecular agent was formerly known as PTC124.   It’s believed that it can assist a lot in   curing certain genetic disorders especially in situations where there’s a mutation of gene.   The molecule has been tested on people living with cystic fibrosis and some other genetic disorders.  Ataluren cystic fibrosis is therefore an attempt to use the tiny molecular agent in curing    cystic fibrosis which has hitherto remained incurable.

So far, the use of Ataluren in treating cystic fibrosis has not yielded   successful results.  The phase 1 of the test yielded no results.  However, the phase 2 trial test carried out among   people living with the disease in Belgium, France and Israel yielded some positive results.   Currently, the phase 3 trial test is on going in the US and other countries in Europe.  It’s expected that the tests will yield better results this time.

Meanwhile some facts are beginning to come up in various sectors concerning Ataluren cystic fibrosis especially online.   One of the latest reports has it that Ataluren is a recent   investigational drug that is being used in the formation of a functioning protein for individual living with genetic disorders as a result of a nonsense mutation. Actually the nonsense mutation refers to the alteration in the genetic code that normally   stops the synthesis of an essential protein.  Currently, the   drug is still under serious tests.  Some medical professionals believe it’s going to be the long awaited cure for cystic fibrosis.

In any case, we still wait to see the results of the tests concerning Ataluren Cystic Fibrosis.  As of now, the disease remains incurable.  It’s purely a genetic disorder that can be inherited.  For a child to inherit the disease, the parents must be carriers of the faulty gene that leads to the disease.   Once the child is born with the disease, the battle for his or her life begins.

In most cases, cystic fibrosis defiles all forms of cure.  It usually comes with several   symptoms   which are seen around the affected organs. The disease   forms thick and sticky mucus around vital body organs such as the lungs, pancreas, liver, intestine and the sweat gland. When the condition or the symptoms are left untreated, serious complications are bound to occur.  The individual involved will suffer untold   health condition in the process.

Well, it’s true that cystic fibrosis still remains incurable; nevertheless, the symptoms can be treated and controlled. Doctors usually engage several tests in arresting the symptoms.  Among such tests include sweat test, blood test, genetic tests and others.  Once the level of the disease is detected through the test, certain medications will be prescribed to treat the symptoms.  While we await further positive results concerning Ataluren,   individuals suffering from cystic fibrosis are encouraged to go for regular medical checkup in order to control the symptoms.

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