8 March 2017

Rare Diseases - The Shock of Diagnosis



It’s very daunting when a member of your family is diagnosed with a rare disease, Something that there isn’t much awareness of, or research about can be really frustrating for you.





That’s how we felt when my partner of 10 years was diagnosed with X – Linked Adrenomyleneuropathy (AMN) Yep, it’s a mouthful right? We weren’t even told during n appointment, we were sent a letter from the neurologist, and discharged from his services because he wasn’t a specialist in it.

I then done something that no one should ever do when in a situation like mine. I hit GOOGLE. And I really shouldn’t have. It took me to the very worst-case scenario and made me so anxious that I didn’t even tell my partner what this diagnosis meant.
We then had to wait for another team of specialists to pick up his case and explain this to us. My anxiety skyrocketed.

AMN is a neurological disease that affects the organs below the waist, The nerve endings protecting the spinal cord were disintegrating making it difficult for the brain to send messages down the spinal cord. In effect, it was stopping his legs from working properly and causing him severe pain. His mobility and balance and quality of life have been severely affected.

This genetic condition is so rare, that even the geneticists and neurological team are working on experience rather than facts, because there is little evidence of what works, or treatments plans available. Each person that is affected by this is completely different. It isn’t textbook stuff and so the medications used to treat his pain, or balance is trailed and discarded at an alarming rate. Thus far he has been through about 6-7 medications with little or no benefit. Because the pain is neurological there really isn’t a huge amount of options, and we are nearing the end of possibilities to help him have a better quality of life.

As if this isn’t enough, our children are also diagnosed with autism and ADHD, Our two girls are obligate carriers for X – Linked Adrenoleukodystrophy (ALD) So they are closely monitored for any changes in their health even though it is considered very rare for girls in childhood to show symptoms of this debilitating disease.

As the whole family seems to be hit by some kind of genetic abnormalities we have been referred for the 100,000 genomes project, a study funded by the NHS that recruits 70,000 patients of rare diseases and cancers to have their whole genomes decoded and analysed to find specific gene mutations. This study is the first and largest of its kind in the whole world, and could ave the way for future diagnoses and treatments of people with rare diseases and cancers and those who are simply named SWAN (Syndrome Without A Name)

I feel privileged to be a part of the part of the future in this study and hopefully one day there will be more research and text book information on the rare disease that my partner sufferers with so that it can help enable people like him to have a better quality of life.

If you are taking part in the 100,000 genomes project then please join the FB Support group






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1 comment:

  1. I can sympathise with your partner having a rare disease myself that effect the myelin on the spine. (PERM) neurological pain is hard to describe and even harder to get rid of :( Id not heard of the 100,000 genome project though, that sounds good. My daughter has just had genetic testing for another rare condition, but I'm not speculating until we get the results xx

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