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To test the in vivo patency of these tissue-engineered veins, two pediatric patients suffering with extra hepatic portal vein obstruction (EHPVO) were transplanted with tissue engineered veins regenerated using 25 ml autologous peripheral blood on compassionate basis.
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Only one patient remains on study drug on a compassionate basis at 12.6+ months.
"We used NIDA marijuana from one of the medical marijuana patients receiving NIDA marijuana on a compassionate basis who sent her marijuana to a DEA-licensed lab conducting our medical marijuana potency study that tested higher potency marijuana from medical marijuana dispensaries," MAPS founder and president told me.
But some also want Sarepta to make the drug available to more patients on a compassionate basis even before approval.
"I have massaged over ten thousand people, and I hope to massage ten thousand more, and to heal the world with good medicine that I can grow here and provide on a compassionate basis to the people who need it".
In 1987, the F.D.A. agreed to allow patients who could not participate in clinical trials — because they were too sick or lived too far from a university hospital, where trials are typically conducted — to obtain experimental drugs from their doctors on a compassionate basis, or as part of a national expanded-access program.
Furthermore we have recently shown that repeated MSC-NTF injections in an ALS patient treated on a compassionate basis were safe and well tolerated[27].
In a recently published case report, it was shown that repeated MSC-NTF injections in an ALS patient treated on a compassionate basis were safe and well tolerated [Muscle Nerve 49 455-457, 2014].
Pentosan polysulphate (PPS), a heparan sulphate mimetic, can prolong incubation time in prion infected mice [34] and is currently being used on a compassionate basis in variant CJD [35], [36].
Meanwhile, pleconaril is still being used successfully on a compassionate basis for treating life-threatening enterovirus infections in children (13 ).
We conducted a retrospective multicenter study of 12 children treated with GO on a compassionate basis (median age 5.5 y).
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Justyna Jupowicz-Kozak
CEO of Professional Science Editing for Scientists @ prosciediting.com