Petition for ALL CHILDREN with nonsense mutation.

[Berit]

Hi all

Please sign this petition to show your support and to urge PTC Therapeutics to provide a new Open Study for ALL children with nonsense mutation using the drug PTC 124.

www.petitiononline.com/ptc124/petition.html


Berit

[David Feder]

blogs.wsj.com/health/2008/12/17/federal-court-denies-teen-experimental-muscular-dystrophy-drug/


Federal Court Denies Teen Experimental Muscular Dystrophy Drug
Posted by Jacob Goldstein



Jacob Gunvalson with his parents.
(AP Photo/The Fargo Forum, Darren Gibbins)
A small drug company doesn’t have to give its experimental medicine to a terminally ill teenager, a federal appeals court ruled yesterday.

The decision overturned a lower court’s ruling from earlier this year, revealing just how complicated this sort of thing can be.

The U.S. Court of Appeals for the Third Circuit in Philadelphia ruled the lower court “abused its discretion” when it ordered PTC Therapeutics to provide its experimental drug PTC124 to Jacob Gunvalson, a 17-year-old boy with a rare genetic called Duchenne muscular dystrophy. Here’s the report from the Associated Press.

Though the particulars vary from case to case, this sort of conflict has become rather common in recent years.

A sick patient without any good treatment options wants to take an experimental drug, but doesn’t qualify for the studies being used to test the medicine. The company that makes the drug typically argues that providing it outside of clinical trials could harm patients in the long run — if, for example, the patient has unexpected side effects that hurt the drug’s chances for approval, or if other patients decide they want access to the drug without enrolling in the clinical trials necessary to determine whether the medicine actually works.

In the case of Jacob Gunvalson, the family has argued that PTC initially led the family to believe that the boy would receive the drug (a claim PTC denies). The family’s attorney told the AP yesterday’s ruling was “a surprise, a huge disappointment, a real tragedy.”

PTC’s president called the ruling important “for the future of the clinical trial process for all experimental drugs for rare disease.”

The lower court’s ruling had been stayed pending appeal, so Gunvalson never received the drug. The family’s legal team is weighing its options.

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[David Feder]

Thank you for signatures in the petition. The origin of subscribers from all parts of the world, you can see that there was a disclosure. I expected greater participation of people. I do not know because it happened. I know many parents have not signed the petition. Why the disinterest in helping? Why the apathy? We are a community? We are all in the same boat? This reminds me the famous quote:
First they came for the Communists,
and I didn’t speak up,
because I wasn’t a Communist.
Then they came for the Jews,
and I didn’t speak up,
because I wasn’t a Jew.
Then they came for the Catholics,
and I didn’t speak up,
because I was a Protestant.
Then they came for me,
and by that time there was no one
left to speak up for me.