When you, your child or loved one is diagnosed with Niemann-Pick disease it can have a profound effect on your whole family. For many people, a diagnosis of Niemann-Pick disease does not happen instantly and can come at the end of weeks or months of tests and investigations.
In many cases this can be the point from which you begin to contemplate the future…
During this period of adjustment you and your family may face what feels like a never-ending round of appointments as you begin to meet with the many professionals who will form a network of care around you or your loved one and you may find yourself having to tell your story time and time again. Hearing the news that your child or family member has a life-affecting disease, but that no one can tell you the exact path the disease will take, may make you feel that your hopes and dreams have been shattered.
At this time there is a lot to think about. Diagnosis comes at a point when you may not be capable of thinking clearly or taking information in.
We have people dedicated to support you to digest all the information:
If you would like to speak to someone about any aspect of the disease:
Kim Stratton, Chief Executive Officer at Orphazyme, commented, “We are delighted with FDA’s decision to grant Breakthrough Therapy Designation to arimoclomol for NPC. Arimoclomol has been shown to have a clinically meaningful effect on disease progression in NPC that is further supported by a biomarker effect indicating an effect on the biological underpinnings of the disease and a favorable safety and tolerability profile. We are committed to bringing this product to patients as soon as possible. Breakthrough Therapy Designation is designed to expedite the development and review of products for serious diseases with the direct involvement of senior staff and we look forward to working closely with the FDA to further advance arimoclomol. Our preparations for filing in the US are underway and we are on track to submit a New Drug Application in H1 2020.”Read more