In a previous study, AMT's gene therapy approach was shown to be successful in the treatment of diseased human muscle cells obtained from biopsies of DMD patients. These data establish a robust basis for AMT's therapeutic approach to DMD. “We are proud to establish proof of concept with our gene therapy for Duchenne muscular dystrophy, as it is an important new step in developing a treatment for this progressive and devastating disease," said Joern Aldag, Chief Executive Officer of AMT. "In particular, our positive results in the heart and respiratory muscles bring hope that we might be able to prevent the fatal outcome of this disease in the future, potentially through a long-term remedy with a single treatment."

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a severe disease characterized by progressive muscle degeneration. It affects young children, almost exclusively boys, and leads to paralysis and death in young adulthood. The disease is caused by mutations in the dystrophin gene, as a result of which the production of functional dystrophin protein, an important structural component within muscle tissue, is blocked. Currently, there is no treatment to prevent the fatal outcome of this disease. DMD affects one in 3,500 males, making it the most prevalent of muscular dystrophies. AMT is developing a gene therapy product for DMD based on 'exon skipping' technology which results in bypassing the genetic defect such that the functional protein can be formed again. Positive long-term therapeutic effects of this approach have been demonstrated in animals.

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT's proprietary platform holds tremendous promise for thousands of rare (orphan) diseases, especially diseases that are caused by one faulty gene. Currently, AMT has a pipeline with nine products at different stages of research or development.

SOURCE: Amsterdam Molecular Therapeutics B.V

The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.

The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.

Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!

https://www.amazon.com/Legacy-Marie-Schlau-collective-Friedreichs-ebook/dp/B01N28AFWZ

 

Research projects currently being financed by BabelFAmily

Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:

1) Gene Therapy for Friedreich's Ataxia research project:

https://www.irbbarcelona.org/en/news/international-patient-advocates-partner-to-fund-spanish-gene-therapy-project-to-treat

The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.

2) Frataxin delivery research project:

https://www.irbbarcelona.org/en/news/new-research-front-to-tackle-friedreichs-ataxia
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.

The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.

 

 

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