Kids with Rare, Deadly Bone Disorder Gain Hope From New Therapy

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Credit: Lindsey Elsaesser

Kids with Rare, Deadly Bone Disorder Gain Hope From New Therapy

When Lindsey Elsaesser wasِ 20 weeks pregnant, anِ ultrasound revealed herِ unborn baby girl hadِ extremely fragile bones.
They thought sheِ would die fromِ respiratory failure becauseِ her bones wereِ soِ weak, Elsaesser said.
When Elsaesser’s daughter, Evie, wasِ born inِ September 2009, doctors wereِ cautiously optimistic aboutِ her condition.

New drug

In theِ study, whichِ included 11 infants andِ children withِ severe hypophosphatasia, theِ drug healed bones, reduced deformities ofِ the skeleton andِ improved children’s strength andِ breathing abilities.
One 3-year-old whoِ wasِ unable toِ stand beforeِ treatment climbed upِ a ladder withِ assistance afterِ two months ofِ therapy.
Previous attempts toِ treat patients byِ transfusing theِ needed enzyme intoِ their blood proved unsuccessful.

Promising treatment

It’s extremely promising, Dr.
David Rimoin, a medical geneticist atِ Cedars-Sinai Medical Center inِ Los Angeles, saidِ ofِ the treatment.
Today, Evie isِ doingِ great, andِ is enrolled inِ music classes, Elsaesser said.