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Re: Miracle Drug for Myotonia????

Hi there. I just want to chime in to support what Jan said about steroids. First, I want to be sure that you understand that the Decadron does not "fix" myotonia. All it does is decrease or control the symptoms. The myotonia will always be there, and I hope you are explaining what it is to your daughter, so that she knows it is not something to be ashamed of or embarrassed about.

Steroids do affect the metabolism, and how and where the body deposits fat, so not only is a diabetic type of diet important, but so is regular exercise. Steroids can also affect blood sugar, blood pressure (sometimes due to water and salt retention) and other issues.

Some people have to be on steroids long term for medical reasons. I would consider, as your daughter learns about her condition and understands it, tapering her off decadron, and putting her in things like karate or tai chi, yoga, and non-competitive sports that she can use to learn the methods of controlling and working with her myotonia. Kids with epilepsy or juvenile diabetes learn about their conditions as young as 4 or 5, and seem to do pretty well with being made "partners" in their own care and destinies.

My personal opinion is that masking the symptoms won't allow her to understand her body as well, and may actually be more difficult on her later on. I was not able to run from a standstill, so I would warm up as I waited. The first few steps were always very challenging, but it made me work harder, and I ran pretty fast for a short kid.

Almost everyone has some thing they are self-conscious about. Myotonia is, at least, not dangerous or progressive - and if you have a good sense of humor and outlook, your life is pretty normal! Lois

Type of Myotonia: MC- Becker's type

Country: USA

Re: Miracle Drug for Myotonia????

Hi,
In my opinion there is nothing to do with sense of humor and disability. If course one can say “I have lost my leg (etc.), but I have gained a new life” , have good mood , sense of humor - but it (disability) still remains not ones choice. Medicines give you a choice.

I admire Lois for good coping strategies with myotonia. But when I was young, I felt very bad among other kids and most of you probably felt the same way. Children can be very cruel if you are different… Coping strategies are good but in many cases not good enough.

Now, when I am 39 it is still difficult for me to think that I could lead “normal” life without mexitil. I am very often depressed because I do realize that mexitil is more and more difficult to get (I ‘m also more tolerant for this drug…). I realize how uncertain my future is. I am also depressed because I remember my childhood… Now, I could try to explain myself many things, but It would not be possible to work like today, play with kids like today.

Julie, if you decide for pharmacotherapy you always have to have someone who is experienced with steroids, who calculates risk/benefit ratio. In case of children mexiletil seem to be much safer choice then steroids. Steroids affect the methabolism but also may cause stop growing up! You may prevent osteoporosis but not stopping growing up (it may be dose dependent). Final thing – it is much safer to take steroids only in the morning and in the afternoon - not at night. It is because of natural rhythm of excreting endogenous steroids.

Observations that steroids (dexamethasone) can alleviate myotonia symptoms are very interesting (and exciting for me) and require further evaluation. It is good to have another therapeutic option. For me this information is like a light in long tunnel, because at any moment anyone on the forum can inform us of “new” medicine for myotonia – medicine that one was taking from other indications that myotonia.
Hugs
Kwak

Type of Myotonia: Thomsen's

Country: Poland

Re: Miracle Drug for Myotonia????

Kwak,

If you'll email me privately I'll send you some information on a medication you might want to try that would be available in Poland.

Jan

Type of Myotonia: Thomsen's

Country: US

Re: Miracle Drug for Myotonia????

Jan I was wondering..... at the dose that my daughter is taking.... 5ml of decadron.... which is 0.5mg of cortisone... is it possible like Kwak was saying for her to stop growing????????????

Type of Myotonia: Thomsen's

Country: Lebanon

Re: Miracle Drug for Myotonia????

Julie,

That is a very low dose but since every person is so different, and children different than adults, I would never be willing to say there would be no side effects at that level. That is basically the only ingredient other than the suspension to make it liquid.

The goal with long-term dexamethasone therapy is to take it once every three days so that the adrenals don't atrophy. That's much more likely with larger doses, but in a child I would want to be very careful.

It's also important for the body to have a period of time (during the night) when inflammatory prostaglandins are increased to repair damaged tissue and to recognize things like early cancer cells and destroy them. For instance women who don't have a natural rise and fall of cortisol are much more likely to get breast cancer. Because of that, like Kwak says, it's best to take a single dose before 9am every morning. (That is what is recommended in the pharmaceutical literature.) You might want to ask your doctor about that, and then see if she gets the same benefits if she drops to once every other day, then once every three days. That can be done over many months, but you want the lowest possible dose to achieve the desired results.

As long as your doctor is monitoring things like blood sugar, bone density, and cortisol levels, I think you'll be fine, especially if it helps her so much. You just don't want to get to a suppressive dose because sometimes the adrenals never start working on their own again. I'm not sure anyone can tell you what that dose would be - it will just have to be monitored.

I'm hoping our pharmacist, Anne, sees this post and will give her input. The only other drug for MC that I would even remotely consider as a parent until teen years would be Diamox (acetazolamide). Since that didn't work for her, then this is certainly the next best thing. I personally know of too many side effects from the anti-seizure and anti-arrhythmic medications in children. They can be wonderful for epilepsy where the benefits outweigh the risks, but for such a benign condition as MC I don't think it's worth it.

Jan

Type of Myotonia: Thomsen's

Country: US

Re: Miracle Drug for Myotonia????

Jan, is it possible for my daughter to stop growing cuz the small amount of steriod that she is taking.... which is 1 mg a day... like kwak said.....
is there any chance she can stop growing with this small amountÉ

Type of Myotonia: Thomsen's

Country: Lebanon

Re: Miracle Drug for Myotonia????

Julie,

I emailed a pediatrician who is an expert on this and will let you know what he says.

Jan

Type of Myotonia: Thomsen's

Country: US

Re: Miracle Drug for Myotonia????

thanks Jan.... you are a huge help... seems like you know everything there is to know.... even more than the docs that we have seen.....

Type of Myotonia: Thomsen's

Country: Lebanon

Re: Miracle Drug for Myotonia????

Julie,

I sent you a copy of this to your email along with the attachments, but wanted to post the information on the forum, too.

If anyone would like the supporting articles let me know and I'll email them to you.

Dexamethasone is apparently a very strong steroid (which contraindicates much of what I had read and is why I try to get an expert opionion!).

Jan


from Dr. Scott Rivkees, Professor of Pediatrics and Director of Yale Child Health Research Center:

Thanks for your note.

Attached are a few reports on the subject.

Dex is very potent in suppressing growth.
Thus low doses need to be used and close follow-up is needed.
With doses too high and not close oversight overtreatment can occur.

For infants we use 0.1 mg per day
For children 0.1 to 0.2 mg
For adolescents 0.25 mg.
AND the doses are invidualized.
Above these doses, growth will slow

0.5 and 1.0 mg doses are high.
These will be associated with weight gain and growth slowdown.
However the treatment goal in the condition you write about may be different.

We give once a day in the AM.
It is twice as potent when given in the evening.
We only use the dilute elixer 0.1 mg/ml and use dosing syringes.

Hope this helps.

Scott Rivkees

Type of Myotonia: Thomsen's

Country: US

Re: Miracle Drug for Myotonia????

Don't forget to consider psychiatric type side effects like depression,mood swings,ect. Last year I was prescribed methylprednisolone packs twice and later had epidurals, with I think dexamethasone in them. These were for herniated discs and not Myotonia and I was amazed at how fluid I was on staircases,and getting up from a chair. It's a tough call you want to be quick from a medicine but you don't want long term damage. I take the Diamox everyday and it works ok. I remember being very frustrated from grade 1-12 with Myotonia and it seemed worse then than it does now.

Type of Myotonia: thomsen's type

Country: USA

Re: Miracle Drug for Myotonia????

I asked my doctor to put me on prednisone since it is the only thing that has made me feel better. He refused but gave a met pack for my up coming vaction. Oh well... The problem is my doctor just wants me to take pain medicine for the rest of my life. I understand the problem with prednisone but take pain pills is not a soltion.