Life just got a little easier for parents of young kids with Tyrosinemia Type I (HT-1)
Raising a child with Tyrosinemia requires constant, attention to diet, a special formula, and drug treatment. The special diet is low in protein and attention to it is critical. Not only do parents have to understand this, but they need to make sure that siblings, grandparents, neighbors, school teachers, camp counselors and others know that restricting your child’s diet is not being mean- it’s showing your love.
In addition to tracking every bit of protein in their kids diet, feeding them a special formula to make sure they get the protein they do need to grow and develop that is free of tyrosine and phenylalanine, is important for normal growth and development.
The third leg, of managing this condition is medication. A medication called Orfadin™ ( nitisinone) blocks the harmful by-products associated with tyrosinemia.
Until this week, the medication Orfadin(nitisinone) came only in capsules, Parents had to open a capsule and remove and weigh a portion of the powder, then mix it with liquid to administer the medication to their infants and young kids. As you can imagine, this is a delicate, time consuming operation. This week Sobi announced that,
“the US Food and Drug Administration (FDA) has as of 22 April 2016, approved Orfadin® (nitisinone) Oral Suspension for the treatment of hereditary tyrosinaemia type-1 (HT-1) in combination with dietary restriction of tyrosine and phenylanine.”
No more breaking open capsules and mixing for infants and young kids! It should be available in August. Thank you Sobi!