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World Hypophosphatasia Day

Hypophosphatasia patients celebrate: "A gateway to a better life has been opened for us."

MedExpress Team

Medexpress

Published Oct. 30, 2024 08:44

Hypophosphatasia patients celebrate:  "A gateway to a better life has been opened for us." - Header image
Thinkstock/GettyImages
This year's celebration of World Hypophosphatasia Day (October 30) will be marked by the joy of patients and their loved ones in gaining access to the first and only targeted therapy for this ultra-rare genetic disease. - Reimbursement of the treatment opens a gateway to a better life - rejoice the parents of children with hypophosphatasia, whose bones break as easily as matchsticks - which is why the condition is called "brittle bone disease."

Enzyme therapy for patients with bone symptoms in the course of hypophosphatasia (HPP for short) has been included in the list of reimbursed drugs effective October 1. Importantly, access to the treatment is available to both children and adult patients with HPP, something both the patients themselves and experts have been calling for.

- Until now, hypophosphatasia has been a disease that has taken away health and life, not to mention dreams. One in three patients, including myself among others, must get around with the help of a wheelchair. Treatment won't fix what the disease has already destroyed, but it can prevent further dramas and open a gateway to a better life for patients," believes Kamila Dratkowicz, president of the Hypogenek Foundation, which works for HPP patients and their f...

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