Monday, September 19, 2016

DMD Treatment approved by FDA

Yesssss,  indeed... the treatment for DMD is now available....

How long have we been waiting.... decades... almost 3; after the discovery of underlying gene mutation called DYSTROPHIN by Louis Kuenkel and his group (1987).

Let us hear the good news from the horse's mouth... FDA report link

But hold on.... it is not available for the entire group of patients suffering due to Duchenne. Presently, it is kind of fix ONLY for those suffering due to mutation of dystrophin gene at Exon location 51.

To be able to follow the above statement; one need to understand that DMD is caused due to mutation in dystrophin gene; which could occur at few notable exon locations i.e,. 12, 13, 17, 19-22, 42-45, 47-53, 55, 60 . How about all those other patients who are having mutations other than exon-51; as a "common man" my guess is...... they just have to wait.... how long??? again... a crude guess is 2-3 years.

At present let us celebrate the moment of joy for all those who are suffering due to exon-51 can knock the doors of the respective state health providers and will have to check out the availability of medicine. I am sure the cost implications are going to be really really huge; as per the press report, Sarepta (the manufacturer) said "the drug would cost about $300,000 dollars a year in USA", this would translate to around TWO crore Indian rupees.

Deep from my heart... I congratulate the entire Muscle Dystrophy community (I being one among them) and today, I am going to have a peaceful sleep... at least for TODAY....

Would be glad to clarify the doubts: smahmedhyd@gmail.com , +91 94 9204 2609 (S.M. Ahmed)