New Drug Practices Improves Life of Fabry Disease Patient

New drugs can often make a huge difference in a patient’s life. Especially in the case of rare conditions like Fabry disease, where there may be a limited number of treatment options. According to a BBC report, new drugs and plans to provide better access to them will dramatically change the lives of many patients. Keep reading to learn more about this development.

Cindy Jones has Fabry disease. Fabry disease is a genetic, lysosomal storage disorder. It results from the build up of a specific fat which the body is unable to process. As it progresses, Fabry disease causes potentially fatal damage to the heart, and kidneys. Sometimes it even affects the nervous system. To learn more about this rare disease, click here.

As Ms. Jones explains in a video segment, she often had to go to the hospital for treatment. She receives infusions of an enzyme as part of her treatment.

Ms. Jones describes this process as a “massive woof.” The treatment is a huge shock to the system. The process leaves her exhausted.

Following the creation of a treatment fund totaling £80m, Cindy Jones will be able to receive a new form of treatment. She will now have access to a tablet version of her enzyme replacement. Furthermore, she will be able to take the tablet at home. Over time, this spaces out the enzyme replacement. Ms. Jones is hopeful that with the new medication and the new schedule some of the side effects of her treatment will be problems of the past.

Things will balance out, Ms. Jones says. She expects it will make a “massive difference” to her quality of life.

While she acknowledges that the new treatment is unlikely to cure her disease, being able to take a tablet every other day at home will make her treatment much more comfortable and manageable. She will likely have to live with the damage already done, but the new medication will be able to stabilize her condition.

With the new fund also came new standards. The Welsh government, for example, set a goal of having new medications available to patients 60 days after approving them. Within six months of setting this goal the time frame was reduced to only 17 days. The average time has been shrinking ever since.

Medications are now made available to patients just 10 days after they are approved by the government of Wales.

82 new drugs have been approved since last year. 20 of these 82 are new drugs targeting cancer.


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