RV: New Duchenne Research Report.
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Georgina Alejandrina Guerrero Torres
Apr 13, 2015, 5:24:49 PM4/13/15
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Estimados, adjunto información de gran importancia la cual resume los avances científicos.
Saludos,
Georgina
-----Mensaje original-----
De: Dr. Guenter Scheuerbrandt [mailto:gscheue...@t-online.de]
Enviado el: lunes, 13 de abril de 2015 11:56 a.m.
Para: Günter, Scheuerbrandt
Asunto: New Duchenne Research Report.
Dear Friends and Colleagues:
This letter goes to you and about 1,400 other Duchenne families and scientists because you had received, often for many years, my reports about research for finding therapies for boys with Duchenne muscular dystrophy. One year ago (January 2014), I sent you a “good bye” letter, because I thought that – at my age of then 83 years – it was time to find someone who could continue my work in a similar way as I had done it. Unfortunately, this does not happen as I hoped for and even my internet pages containing many of my older reports and interviews (www.duchenne-information.eu) have been taken away and I do not know how they can be used by me again.
Just at this time, when exon skipping, the most advanced Duchenne research approach, has encountered serious difficulties, it is unfortunate that my reports are not available anymore which I had written since the year 2000 especially for Duchenne families. I therefore thought a few months ago that I should try to find out whether you and many others who are receiving this letter would like me to continue my reports, but perhaps concentrating first on exon skipping and later on other techniques also, which are not too far from becoming approved as effective treatments.
Thus, I have written the enclosed rather short report only about the work done by the Dutch company Prosensa on exon skipping during the last two years after the failure of the phase-III clinical trial with drisapersen to skip exon 51 of the dystrophin gene. You know from my last report, written at the beginning of 2013, that this trial was the “pivotal”, the decisive one, because it was designed to definitely prove that exon skipping really is able to slow down the degeneration of Duchenne muscles. We all had hoped that this trial would really show the results that would soon lead to the effective and approved skipping drugs we are waiting for. Unfortunately, on 20 September 2013, it became publically known that the results of this trial were not significant. Thus, in the last two years, Prosensa, which was left alone by its large partner GlaxoSmithKline, started to find by itself the reasons of this failure and it still continues that, but now as part of the American company Bi oMarin near San Francisco which has much experience with developing therapies for rare diseases.
But after this short updated exon skipping report you might be interested to follow for yourself on the internet the new advances of important Duchenne research. So you may ask the following selected companies and patient organizations to send you regularly their press releases or newsletters.
Two companies are working on exon skipping, Prosensa-BioMarin in Leiden, the Netherlands, (www.prosensa.eu), and Sarepta in Cambridge in the United States (www.sarepta.com). The PTC company in New Jersey in the USA (www.ptcbio.com) has now received permission to market their drug Translarna (earlier called PTC124 and Ataluren), for reading through premature stop codons, which, however, can be used only by patients, who have these stop codons produced by some special point mutations. There are also two other quite advanced research approaches for developing Duchenne drugs that are independent of the patient’s mutation. The company Summit near Oxford (www.summitplc.com) develops the potential drug SMT C 1100 for the upregulation of utrophin, and Santhera near Basel in Switzerland (www.santhera.com) works on Catena/Raxone for slowing the degradation of respiratory function in older patients. The parent association Parent Project Muscular Dystrophy in the USA (www.parentprojectmd.org) publishes regularly summaries about practically all research work on treating Duchenne muscular dystrophy. And also our European center for neuromuscular diseases in Newcastle upon Tyne in England (www.treat-nmd.eu) publishes newsletters regularly. All the information on these websites is in English. And there are many other associations and companies working on Duchenne research which I cannot mention here.
I am hoping that many of you who are receiving this letter will let me know what you think of my starting again my reports for as long as I am not too old for doing that. With kind regards, I am your now almost 85 year old friend Guenter.
Guenter Scheuerbrandt, PhD.
gscheue...@t-online.de
Im Talgrund 2, 79874 Breitnau
Germany,
14 April 2015
PS: I am not charging you anything for this new report. However, if you would like to help me with the not so easy private financing, I have a special bank account for this. But please mention your e-mail address so that I can say thank you. GS.
IBAN: DE29 6805 1004 0004 6026 32, BIC: SOLADES1HSW
Saludos,
Georgina
-----Mensaje original-----
De: Dr. Guenter Scheuerbrandt [mailto:gscheue...@t-online.de]
Enviado el: lunes, 13 de abril de 2015 11:56 a.m.
Para: Günter, Scheuerbrandt
Asunto: New Duchenne Research Report.
Dear Friends and Colleagues:
This letter goes to you and about 1,400 other Duchenne families and scientists because you had received, often for many years, my reports about research for finding therapies for boys with Duchenne muscular dystrophy. One year ago (January 2014), I sent you a “good bye” letter, because I thought that – at my age of then 83 years – it was time to find someone who could continue my work in a similar way as I had done it. Unfortunately, this does not happen as I hoped for and even my internet pages containing many of my older reports and interviews (www.duchenne-information.eu) have been taken away and I do not know how they can be used by me again.
Just at this time, when exon skipping, the most advanced Duchenne research approach, has encountered serious difficulties, it is unfortunate that my reports are not available anymore which I had written since the year 2000 especially for Duchenne families. I therefore thought a few months ago that I should try to find out whether you and many others who are receiving this letter would like me to continue my reports, but perhaps concentrating first on exon skipping and later on other techniques also, which are not too far from becoming approved as effective treatments.
Thus, I have written the enclosed rather short report only about the work done by the Dutch company Prosensa on exon skipping during the last two years after the failure of the phase-III clinical trial with drisapersen to skip exon 51 of the dystrophin gene. You know from my last report, written at the beginning of 2013, that this trial was the “pivotal”, the decisive one, because it was designed to definitely prove that exon skipping really is able to slow down the degeneration of Duchenne muscles. We all had hoped that this trial would really show the results that would soon lead to the effective and approved skipping drugs we are waiting for. Unfortunately, on 20 September 2013, it became publically known that the results of this trial were not significant. Thus, in the last two years, Prosensa, which was left alone by its large partner GlaxoSmithKline, started to find by itself the reasons of this failure and it still continues that, but now as part of the American company Bi oMarin near San Francisco which has much experience with developing therapies for rare diseases.
But after this short updated exon skipping report you might be interested to follow for yourself on the internet the new advances of important Duchenne research. So you may ask the following selected companies and patient organizations to send you regularly their press releases or newsletters.
Two companies are working on exon skipping, Prosensa-BioMarin in Leiden, the Netherlands, (www.prosensa.eu), and Sarepta in Cambridge in the United States (www.sarepta.com). The PTC company in New Jersey in the USA (www.ptcbio.com) has now received permission to market their drug Translarna (earlier called PTC124 and Ataluren), for reading through premature stop codons, which, however, can be used only by patients, who have these stop codons produced by some special point mutations. There are also two other quite advanced research approaches for developing Duchenne drugs that are independent of the patient’s mutation. The company Summit near Oxford (www.summitplc.com) develops the potential drug SMT C 1100 for the upregulation of utrophin, and Santhera near Basel in Switzerland (www.santhera.com) works on Catena/Raxone for slowing the degradation of respiratory function in older patients. The parent association Parent Project Muscular Dystrophy in the USA (www.parentprojectmd.org) publishes regularly summaries about practically all research work on treating Duchenne muscular dystrophy. And also our European center for neuromuscular diseases in Newcastle upon Tyne in England (www.treat-nmd.eu) publishes newsletters regularly. All the information on these websites is in English. And there are many other associations and companies working on Duchenne research which I cannot mention here.
I am hoping that many of you who are receiving this letter will let me know what you think of my starting again my reports for as long as I am not too old for doing that. With kind regards, I am your now almost 85 year old friend Guenter.
Guenter Scheuerbrandt, PhD.
gscheue...@t-online.de
Im Talgrund 2, 79874 Breitnau
Germany,
14 April 2015
PS: I am not charging you anything for this new report. However, if you would like to help me with the not so easy private financing, I have a special bank account for this. But please mention your e-mail address so that I can say thank you. GS.
IBAN: DE29 6805 1004 0004 6026 32, BIC: SOLADES1HSW
maria mendez
Apr 23, 2015, 11:49:44 AM4/23/15
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disculpe esto esta en español.
Saludos,
María José
Saludos,
María José
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> ---
> Has recibido este mensaje porque estás suscrito al grupo "UPA! Cura Duchenne" de Grupos de Google.
> Para anular la suscripción a este grupo y dejar de recibir sus mensajes, envía un correo electrónico a dmdla+un...@googlegroups.com.
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> --
> Usted recibió este mensaje porque usted está suscrito al grupo de Google "UPA! Cura Duchenne". Para postear un mensaje, envíe un mail a DM...@googlegroups.com.
>
> Para eliminar su suscripción de este grupo envíe un mail a la siguiente dirección: DMDLA-un...@googlegroups.com
>
> Si desea ir a la página de UPA! Cura Duchenne - vaya a www.upaduchenne.org
> Para más opciones, visite el grupo en: http://groups.google.es/group/DMDLA?hl=es
>
> ---
> Has recibido este mensaje porque estás suscrito al grupo "UPA! Cura Duchenne" de Grupos de Google.
> Para anular la suscripción a este grupo y dejar de recibir sus mensajes, envía un correo electrónico a dmdla+un...@googlegroups.com.
> Para obtener más opciones, visita https://groups.google.com/d/optout.
Marisa Rodriguez
Apr 23, 2015, 1:48:15 PM4/23/15
to dm...@googlegroups.com
DEAR Gunther: IF YOU NEED TO CONTINUE WITH THESE IMPORTANT CONTRIBUTIONS OF PROGRESS.
I WILL APPRECIATE IN DEPTH.
THEM IF POSSIBLE IN SPANISH ALSO WOULD BE VERY USEFUL. IF WE TRANSLATING BY GOOGLE.
THOUSAND THANKS
YOU STILL WAITING
I WILL APPRECIATE IN DEPTH.
THEM IF POSSIBLE IN SPANISH ALSO WOULD BE VERY USEFUL. IF WE TRANSLATING BY GOOGLE.
THOUSAND THANKS
YOU STILL WAITING
MARISA
De: maria mendez <peluss...@hotmail.com>
Para: "dm...@googlegroups.com" <dm...@googlegroups.com>
Enviado: Jueves, 23 de abril, 2015 12:19:42
Asunto: RE: {UPA!} RV: New Duchenne Research Report.
Enma Morales
Dec 11, 2015, 5:54:39 PM12/11/15
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Dios les bendiga siempre y felices fiestas de fin de año y Dios les multiplique fuerzas,salud y bendiciones por tan noble labor.
Enviado desde mi Samsung Mobile de Claro
Enviado desde mi Samsung Mobile de Claro
-------- Mensaje original --------
De: maria mendez <peluss...@hotmail.com>
Fecha:23/04/2015 09:49 AM (GMT-06:00)
A: dm...@googlegroups.com
CC:
Asunto: RE: {UPA!} RV: New Duchenne Research Report.
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