Comments on: Stem cell hope for Duchenne muscular dystrophy

By: Paul Browne

Paul Browne — Wed, 09 Nov 2011 11:04:31 +0000

Amit, there is quite a lot of research under way into muscular dystrophy, particularly Duchenne Muscular Dystrophy.

The exon 51 skipping approach that Anup mentions is one promising new therapy that is already being evaluated in clinical trials, as we discussed in a post earlier this year http://speakingofresearch.com/2011/08/01/promising-clinical-trial-result-for-exon-skipping-in-duchenne-muscular-dystrophy/ while an earlier post described a modified version of this approach that may be applicable to DMD patients with mutations in other exons http://speakingofresearch.com/2009/03/23/progress-towards-a-cure-for-duchenne-muscular-dystrophy/

Hope this is helpful for you!

By: Anup

Anup — Sun, 06 Nov 2011 15:33:56 +0000

Amit,
Exon skipping is a promising research it seems but then that is for DMD. You have not mentioned whether you are looking for DMD or some other muscular dystrophy. Also, which are the exons deleted? Exon 51 skipping is at most advanced stages of research.
Rgds….Anup

By: Tom

Tom — Sun, 06 Nov 2011 15:07:48 +0000

There is a huge amount of research coming through, but it often takes years for it to actually reach the markets – something scientists and regulators are trying to speed up.

By: Amit Sharma

Amit Sharma — Sun, 06 Nov 2011 14:04:13 +0000

Is there any recent research going on muscular destrophy. Is there any treatment till now. Please send the reply quickly.

By: Blue Sky Science

Blue Sky Science — Sat, 12 Feb 2011 12:50:18 +0000

I’m sorry to hear about your son’s DMD, and admire your decision to see if he can take part in a DMD therapy trial, such trials make a vital contribution to the development of medicines which may benefit not only your own son but thousands of other people with muscular dystrophy. I’m not qualified to give professional advice on participation in clinical trials, but it is clearly important to ensure that any trial your son participates in is appropriate for him.

The UK Muscular Dystrophy Campaign has some good general advice at:

http://www.muscular-dystrophy.org/research/your_questions_answered

The US National Institutes of Health (NIH) has some information, with links to useful resources on clinical trials at:

http://www.ninds.nih.gov/disorders/md/md.htm#Clinical_trials

A good place to start is the ClinicalTrials.gov website, which lists all clinical trials that have been registered in the US, and many in other countries, I searched using the keywords “muscular dystrophy” and limited my search to active trials, and found 47 trials which are either recruiting patients or will soon start to do so.

http://clinicaltrials.gov/ct2/results?term=Muscular+Dystrophy&recr=Open&rslt=&type=&cond=&intr=&outc=&lead=&spons=&id=&state1=&cntry1=&state2=&cntry2=&state3=&cntry3=&locn=&gndr=&rcv_s=&rcv_e=&lup_s=&lup_e=

At the risk of stating the obvious, I would also add that before taking part in a trial you should discuss the trial thoroughly with your son’s consultant, and make sure that all relevant medical records are available for the clinical trial team to examine. Make sure that you find out from the clinical trial team who will be responsible for any longer-term follow up care that your son after the trial has officially finished, and how participation in the trial will affect medical insurance cover.

Finally, I would urge you to be very cautious about any clinical trial where you are expected to pay for the cost of the procedure, or for a substantial part of the costs, such charges are often a sign that the trail is not a genuine trial but rather a cover for unscrupulous doctors who want to make money from an procedure which has not yet been shown to work.

I hope this information is useful, and hope that you find a treatment that helps your son.

By: Anup Kalra

Anup Kalra — Sat, 12 Feb 2011 05:03:45 +0000

Hi,

My son is 5 years old is just now diagnosed with DMD. He is ok right now. Have approached for enrolling him for clinical trials with researhers and pharmaceutical companies. Will highly appreciate your guidance in this regard.
Regards…Anup

By: Paul Browne

Paul Browne — Mon, 17 May 2010 10:20:22 +0000

Jyotirmai, I am not a medical doctor so I can’t offer any specific advice, but I do encourage you to discuss the possibility of your son taking part in clinical trials of new treatments with your son’s doctors.

They can advise you on whether there are any clinical trials in progress or being planned that your son could take part in.

You might find the following websites helpful, and they may be able to tell you about clinical trials that are being planned.

http://www.muscular-dystrophy.org/
http://www.mdausa.org/

I hope that your efforts on behalf of your son are successful.

By: Jyotirmai Bartaria

Jyotirmai Bartaria — Tue, 05 Jan 2010 17:32:30 +0000

My 7 year old son is suffering from dmd.I come to know about 4 years before, In begining he didnot climb but walk run easily. But from 3 month before he is unable to stand now he cannot sit self. In dystrophin gene test a deleation of number 12 is told.I want to know any stem cell therepy or medicine can cure him.Thanks.

By: Sarona Stanley

Sarona Stanley — Mon, 14 Dec 2009 04:41:20 +0000

My son was diagnosed with DMD early 2005. He has been wheelchair-bound since. He was 8 years old when he was diagnosed with DMD. He is now 12 years old and the rapid muscle wasting has worsened in the last 3mths. His eyesight has also gone bad in the last 6 months. I wish and pray that the medical profession would come up with some form of stem cell treatment soon for DMD like they did with the 19-year old with Multiple Scloresis earlier today. The Announcement has given all DMD sufferers hope. May our good Lord above continue to guide the medical profession in expediting the process of stem cell treatments for all DMD sufferers including my precious son. Please help make my son live.