Comments on: PTC124, a Drug Against Genetic Diseases: Overview

By: r

Sun, 27 May 2012 17:35:45 +0000

When ptc124 medicine come in market?

By: N.Gandhi Prasad

N.Gandhi Prasad — Thu, 01 Sep 2011 13:57:12 +0000

hi when ptc 124 medicine is available in the market can tell me about it. dis my mail id: gandhi.mca02@gmail.com
pls tell i am suffering MD from 6years so kindly tell me information about PTC124

By: N.Gandhi Prasad

N.Gandhi Prasad — Sat, 27 Aug 2011 16:10:41 +0000

i want ur medicine PTC124 because i am suffering from MD from last 6 years

By: SUDHAKARA REDDY.G

SUDHAKARA REDDY.G — Fri, 19 Mar 2010 06:11:35 +0000

Respected Sir,
My son (Master G.Venu Gopal Reddy ) has been suffering from Duchene Muscular Destrophy It has no treatment .But I felt very happy When I saw your blug. I would like to know the some information such as 1) How long does it take to come to the patients ?
Thank u sir
Yours truely,
G.SUDHAKARA REDDY School Teacher
H.No : 21/ 40 f Balakonda hall street
Nandyal ( Town ) Andhra Pradesh ( State ) India

By: jack

jack — Wed, 10 Mar 2010 22:17:20 +0000

I’m Iranian. duchenne one of my relatives has the disease. In our country for this disease is no treatment. But as far as I saw the Americans found the drug for this disease. I request that you support our oppressed people And poor in this country do not let our beloved bloodthirsty go of my hand.

By: Lindsey

Lindsey — Wed, 10 Mar 2010 03:07:31 +0000

Nice post. scienceroll.com deserves an oscar.

By: shashank

shashank — Sun, 17 Jan 2010 19:29:55 +0000

Here is a link to more information about the genetics of X-Linked Spondyloepiphyseal Dysplasia Tarda that was prepared by our genetic counselor and which has links to some useful resource for those dealing with this condition: http://www.accessdna.com/condition/_X-Linked_Spondyloepiphyseal_Dysplasia_Tarda/677. I hope it helps. AccessDNA

By: Feng Feng

Feng Feng — Tue, 05 Jan 2010 19:20:32 +0000

The pivotal trial of ataluren (formerly known as PTC124 ) in Duchenne’s muscular dystrophy has been completed and top-line pivotal data are expected in the first half 2010. If the data are positive, PTC could be on track to file a new drug application for ataluren in mid-2010 and potential accelerated approval in the first half of 2011. [Reference: http://www.bioworld.com/servlet/com.accumedia.web.Dispatcher?next=bioWorldHeadlines_article&forceid=53098%5D

A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation.

By: Anonymous

Anonymous — Tue, 05 Jan 2010 02:59:49 +0000

What is this suppose to do for kids with Hurler Syndrome? What about if they have been transplanted or not?

By: JYOTIRMAI BARTARIA

JYOTIRMAI BARTARIA — Mon, 04 Jan 2010 09:29:48 +0000

i want to know ptc124 when come in market and who and where i get it my 7 year old child suffering dmd

By: shashankgupta225

shashankgupta225 — Fri, 01 Jan 2010 16:21:15 +0000

Symptoms & Characteristics
X-linked spondyloepiphyseal dysplasia tarda is a genetic condition that impairs bone growth and occurs almost exclusively in males. The name of the condition indicates that it affects the bones of the spine (spondylo-) and the ends (epiphyses) of long bones in the arms and legs. “Tarda” indicates that signs and symptoms of this condition are not present at birth, but appear later in childhood, typically between ages 6 and 10.

Males with X-linked spondyloepiphyseal dysplasia tarda have skeletal abnormalities and short stature. Affected boys grow steadily until late childhood, when their growth slows. Male adult height ranges from 4 feet 10 inches to 5 feet 6 inches. Individuals with X-linked spondyloepiphyseal dysplasia tarda have a short trunk and neck, and their arms appear disproportionately long. Impaired growth of the spinal bones (vertebrae) causes the short stature seen in this disorder. The spinal abnormalities include flattened vertebrae (platyspondyly) with hump-shaped bulges, progressive thinning of the discs between vertebrae, and an abnormal curvature of the spine (scoliosis or kyphosis). Other skeletal features of X-linked spondyloepiphyseal dysplasia tarda include an abnormality of the hip joint that causes the upper leg bones to turn inward (coxa vara); a broad, barrel-shaped chest; and decreased mobility of the elbow and hip joints. Arthritis often develops in early adulthood, typically affecting the hip joints and spine.

By: Hans Lindh

Hans Lindh — Wed, 09 Dec 2009 08:41:04 +0000

Does PTC 124 have effect on X-linked spondyloepiphyseal dysplasia tarda? I understand that it can be nonsense mutations involved i that Genetic Disease as well.

By: Feng Feng

Feng Feng — Wed, 07 Oct 2009 20:53:26 +0000

PTC124 is scheduled to enter the final phase2b of clinical trials in 2009.

Please see the link below for the clinical trials.

http://www.clinicaltrials.gov

By: Feng Feng

Feng Feng — Wed, 07 Oct 2009 20:48:14 +0000

However, it is not equally effective with every stop codon, working best on the sequence ‘UGA’ [1] – PTC124 WIKI

By: MAHESH

MAHESH — Thu, 12 Mar 2009 11:00:10 +0000

hi ptc 124 clinical trials finished. it may come 2 market wth a week.

By: MAHESH

MAHESH — Tue, 03 Mar 2009 14:16:30 +0000

Hi This message for All Indian peoples who are all suffering from muscular dystrophy(different types).

dear friends here a hppy news that the scientists discovered the medicine ptc 124 . still the clinical trials are going on. Eventhough these pills are avaailable in USA and UK. So why cant it is not possible to get in India. Plese try to publish the matter in Daily news papers.

With hope,
Mahesh

By: MAHESH

MAHESH — Tue, 03 Mar 2009 14:13:14 +0000

Hi friens,

upcourse ptc124 is available in U.SA and in U.K . I too dontknow howto get these medicines. Its very beneficial for all type of muscular dystrophy.

Yours faithfully
MAHESH

By: Abdoul Hossein Niksirat

Abdoul Hossein Niksirat — Mon, 09 Feb 2009 17:38:29 +0000

dear sir ,
Please give further information about the medicin PTC124 (inclding
price and how to buy from my country Iran)
Thanking you in anticipation of your kind attention.
Yours faithfully
Abdoul hossein Niksirat

By: K.K.Sharma

K.K.Sharma — Mon, 25 Aug 2008 21:44:43 +0000

Kythryn Wright.
Thanks for the info about PTC124. After long search and talking to several well known neurologists/proffessors. It is clear this medicine is not for polio or PPS. My friends have wasted large sum of money on stem cells if anybody have any info let us know or had any body any beneficial effects.

By: Bertalan Meskó

Bertalan Meskó — Thu, 24 Apr 2008 06:08:54 +0000

It seems it would work with all nonsense mutations.