Tuesday, June 24, 2014

DMD Treatment - Available

Update... 27th August, 2015
Here is the pleasant news FIRST TIME EVER to all those suffering from Duchenne MD and have mutation at the location: Exon-51 in the dystrophin gene. FDA has considered the "New Drug Application NDA for the Eteplirsen drug made by Sarepta Therapeutics.

Details: LINK 

Update... 4th December, 2014
Wake up... GERMANY.... the DMD drug has now been released commercially today (4th Dec., 2014) in Germany to treat DMD patients of 5-years and above, ambulant for a specific mutation.
here are the detailed reports : LINK-1 , LINK-2

The title of this post is certainly going to bring a  "colorful"  ray of hope among so many patients and their families. YES,  THE APPROVAL FOR THE DRUG TREATING DMD HAS BEEN GRANTED ***. The purpose of choosing this title is to bring out the latest news which I have been reading: "The treatment options for Duchenne Muscle Dystrophy, DMD". This can easily be done by all those google users by setting their google alerts with the titles "DMD",  "DMD Treatment". This is how I get to know every whisper on DMD which may happen anywhere around the globe. I am NOT an expert, but a father of patient who is suffering from another kind of muscular dystrophy (LGMD). Let me start with the best news which flashed exactly a month ago (24th May, 2014) about a conditional approval of DMD treating drug, followed by few more medicines which are very very promising and have been going through a thorough grind under the watchful eyes of FDA (Food and Drug Administration), USA and are almost hoping for a green signal.

European Medicine Agency (CHMP, Committee for Medical Products for Human use) recommends first-in-class medicine for treatment of DMD:
Here is the exact report which was declared by European Medical Agency. Without making it any sensational; let me use the exact phrases verbatim : "the CHMP has recommended granting a conditional marketing authorisation for Translarna (Ataluren)" marketed by I. PTC therapeutics USA. Translarna is to be used for in patients aged FIVE years and above who are able to walk.

Even though the regulating agency CHMP had turned down the application of Translarna in January-2014; the present stand is all due to a request of the applicant to re-examine of its opinion on all the available evidence, re-analysis of the clinical data. As per the present stand of CHMP a conditional marketing has been awarded to PTC. This is a marketing authorisation for a drug that addresses an unmet medical needs of patients suffering due to life threatening disease.

II. Prosena gets a FDA green signal
Another report appearing on 3rd June, 2014 sounds a very positive note by saying that Prosena therapeutics, Netherlands has got the favoring (GREEN) signals from FDA. Prosena's drug, Drisapersen too had its flip side when their phase-III trials had not shown appreciable results as compared to the placebo group. However, due to the relentless efforts of the company with new analysis and extension studies on phase trials the company has come under the consideration of FDA. Prosena was asked (by the FDA) to two post approval studies  to confirm drisapersen's efficacy.

III. Sarepta surges ahead on optimism over Muscular Dystrophy Drug
This report mentions the firmed up plans of Sarepta's drug Etiplersen to be getting ready soon. The upbeat mood was all due to the favorable decision the company may hope to get from FDA for moving quickly to the new set of trials with a large group of patients to substantiate the already existing data with a small group of patients.

*** NOTE:
* Most of the information given above is taken from the material appearing in the internet. 
* Credit has been given to all the original reports by providing the link (in bold-red color). 
* The authenticity of the above provided information can be sought from the appropriate drug companies while the  author takes no responsibility. 
* It must also be kept in mind that 
a. the availability of this drug in the market is going to take some time
b. also, the drug could be given only to those patients who will qualify as per the exact mutation for which these drugs have been designed; further details are beyond the scope of this report.

Important link for Calpainopathy patients: LGMD-2A
 Coalition to Cure Calpain, 3:  link
Here is the place where the co-founders of the organisation themselves are suffering due to Calpainopathy and they are taking every possible step which will inch towards the cure of another rare disorder.

Would be more than glad to advice (and help) any one suffering due to this disease:
contact : smahmedhyd@gmail.com 

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