Family Support >>>

Family Surveys
 
Association surveys families on drug benefits
 
April, 2003
 
The NBIA Disorders Association mounted an e-mail survey in January after a family requested information about Sinemet, a drug normally used to treat people with Parkinson’s disease for tremors. The association is grateful to the families who responded and is sharing the results, which are not scientific but could be helpful when discussing approaches with patients’ doctors.

Of the families who responded, eight NBIA patients, including two girls in the same family, reported no improvement from taking Sinemet for relief of any NBIA symptoms. Those patients stopped taking the drug.

In three other patients, improvements were reported. Of those, one said the drug helps but that when too much is given, the result is “dancing legs,” (involuntary movement of the legs). Another said the Sinemet helps muscle spasms best when given with Baclofen. The third patient, who has been on the drug for a year, said it helps with tremors and dystonia. He takes 25/100 mg 3 times a day and said it works best during the day when he is awake.

Another patient in the survey became so heavily sedated by the drug he stopped taking it. That patient, however, may try it again. Another felt there was a decrease in muscle spasms and that it helped, but the side effects of dyskinesia and nausea were overwhelming and outweighed the benefits. The drug was stopped after two weeks.
 
 
Botox survey gets positive, negative feedback
 
Dec. 2002

It’s too early to tell how effective botox treatment is for HSS patients overall, but HSSA has received some feedback on the treatment from families it surveyed over the summer.

After sending a questionnaire about various treatments, board member Kris McGourthy received 12 responses from families who indicated they had tried botox injections.

Nine families said they had positive results at various injection sites: the tongue, arm, neck, jaw, calf and leg muscles. The families reported less stiffness in the muscles, making it easier for the patients to move and get around.

Others had less success. One individual reported a negative reaction to injections in the neck area. That person felt better a few days after the shots but would later “crash and be worse than before.” Two individuals said they did not detect any response to the injections.

As a result of this questionnaire, Kris McGourthy took her son, Michael, for his first dose in July. The dosage was 300 units to his legs. Kris feels they had positive results, in that Michael could walk easier, wasn’t up on his toes as frequently and could get back down when he did go up on his toes. Speech also was improved, even without injections to that area. He recently went for the second time, using 500 units of the botox injection. Kris said the results lasted about three months.

In response to other treatments asked about in the questionnaire, one person reported positive results from using a baclofen pump but later had complications and many follow-up visits. One patient tried a phenol block and had six months of positive results. But that patient also tried a low dose of botox without any benefit.

HSSA wants to do another questionnaire regarding Electrical Muscle Stimulators, acupuncture, chiropractors, spinal manipulation and other techniques families have tried. HSS families should look for this survey in the next month or so. If you do not receive a questionnaire and would like one, please let us know by phone (619) 588-2315 or e-mail info@nbiadisorders.org so we may include you in our results.