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Re: MNTA's M294 -- early/expanded access?

Plasma product shortage has Gilbert woman on edge - 8 July 2019

Excerpts:

“There are only two brands left that they can get and there is no guarantee that these meds will be consistently available for the foreseeable future,” White said. “I’m terrified. I’m absolutely terrified.”

“The different brands are formulated differently,” she said. “Some I can’t tolerate at all.”

Without treatment, White said, “I could end up with pneumonia and my body will succumb from not fighting off the illness.”

Intravenous immunoglobulin or IVIG is used for the treatment of other diseases, including Kawasaki disease, Guillain-Barre syndrome and lupus.

“Immunoglobulin and other plasma products are being used more and more for off-label and investigatory reasons,”

Drugs derived from human plasma take nearly a year to produce, not like a pill where a company can add an extra shift of workers and churn out product

We use it for antibody replacement. We don’t make antibodies so we get immunoglobulin. We are going to get sick, we are going to get an infection a normal person can fight off with their antibodies. We require that protective level of immunoglobulin to stay alive.”

“Everyone in the medical community has to be very thoughtful and they may not have realized the supply was so tight but now that they are dealing with that, it can’t be business as usual,” he said. “They have to think about people who need it the most. “

There is no cure, instead patients like White are treated with immunoglobulin replacement therapy, which most often relieves symptoms. The therapy must be given regularly and life-long.

“All we can do is encourage people to donate plasma,” White said. “It’s scary to think a baby with Kawasaki disease can’t get it and needs it to save his life.”

http://gilbertsunnews.com/plasma-product-shortage-has-gilbert-woman-on-edge/