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August 1996 Newsletter


Research on myotubular myopathy has really picked up! A gene has been located by researchers in Europe. This is probably not the only gene responsible. In fact different cases my be due to mutations in different genes. In the newsletter is an interview with Dr. Gail Herman about this finding. Also, attached is the article announcing the discovery of the gene. (Hope you understand it better than we did!) [Note to our friends on the WWW. This is not included on our site. But we can help you get a copy if you are interested.]

As researchers collect more information about this disease, they are noticing more and more interesting things. For example, a second child has been diagnosed with Spherocytosis, a rare form of anemia caused by a structural defect in the red blood cell. Also, many kids have gall stones, elevated liver enzymes and other unusual disorders.

The occurrences of all these other disorders are too rare to be unrelated. That is why researchers need more information from you and your families. Attached is a clinical questionnaire and release so that Dr. Herman can gather some of this information. Also, we've attached a reminder of some information specific to your family that Dr. Herman also needs. Please mail the release and questionnaire back to Dr. Herman in the enclosed envelope as soon as possible. Also include a spare photo if you have one. Your cooperation may mean the difference in what we learn about Myotubular Myopathy. Also, remember that research like this takes time. Breakthroughs usually take years, they rarely happen overnight.

What does this mean for our kids today? Given all the unusual disorders showing up, Dr. Herman recommends a number of tests. Consider discussing the following with your son's doctor:

  • An osmotic fragility test (the test for Spherocytosis) -- age 6-12 mos. (If Spherocytosis is found, then physicians should not worry about a mild increase in bilirubin.)
  • CBC, Chem 20 -- annually.
  • Bone age x-ray -- by age 3-5 (by age 3 if the child is growing faster than the 95th percentile, by age 5 if growth is normal).
  • Blood pressure -- checked regularly.
  • Hormone tests -- she is still sorting out the correct ones. However, any child with increased bone age, early signs of puberty, history of liver problems, ulcers, bleeding or vitamin K deficiency should get their adrenal/male hormones checked. We'll pass on more about the proper tests in the next newsletter. In the meantime, call us if you are contemplating doing this.

On the advice of some other parents, we recently started giving ... the nutritional supplements Co-Enzyme Q-10 and L-Carnitine. These supplements, available at most health food stores, are involved in the body's process of converting food to energy. Everyone using them seems to agree that their kid's energy levels are increased. We've included some information on both Co-Enzyme Q-10 and L-Carnitine. To be on the safe side, however, please discuss the matter with your physician before starting these or any other supplements. If you do use them, please let us know whether or not you feel they are helpful.

The resource group was recently featured in the July edition of Exceptional Parent magazine. We enclosed a copy of the article. [Note to our friends on the WWW. This is not included on our site. But we can help you get a copy if you are interested.]

On a sad note, we would like to mention the passing of a few special kids since our last newsletter. Our sympathy goes out to the families of [those four children]. Although they remain in our hearts, they are still missed, especially by their families. Please keep them in your thoughts and prayers.

We will keep everyone posted on the progress of the research. We are sensing a real excitement in the research community about all the recent findings. Please help them build on this excitement by providing them with as much information as you can. Also, please remember, these things take lots of time.

Until next time, take care and stay in touch.

 

 

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Copyright © 2000 by the Myotubular Myopathy Resource Group, Inc . Information on this website may be redistributed and copied freely provided that proper attribution is given.  This page was last revised on 15 Feb 1999. The Myotubular Myopathy Resource Group, Inc. is a not-for-profit organization under Section 503(c) of the IRS Code. These World Wide Web pages are published by the Myotubular Myopathy Resource Group, Inc. solely as a service for interested parties. This is a lay interpretation and should not be considered definitive by any means. This discussion draws largely from the sources cited as well as the personal observations of the authors. We are neither doctors nor experts nor do we pretend to be. Any decisions on medical treatments, interventions, courses of action, etc. should be made by the appropriate family members in consultation with the available literature and qualified medical professionals. Good sense should always prevail. The authors, contributors and the Myotubular Myopathy Resource Group, Inc. assumes no responsibility for the use of the information, observations or opinions presented herein.