The study outlined in this notice is being conducted in the United States, but does not require participants to travel. 

The study requires medical records in English.

 

 

----- Original Message -----

From: Linda Condon

To: fapg

Sent: Sunday, November 02, 2008 10:36 PM

Subject: [FAPG] FA cardiac study still recruiting

 

The Children’s Hospital of Philadelphia Announces a Study of Cardiomyopathy in

Friedreich Ataxia Patients

 

Who Can Participate?

 

- Any individual with a diagnosis of Friedreich ataxia (both living and deceased).

 

Why and What Are We Studying?

 

- We are interested in collecting all available cardiac medical records, ECHO tapes, Holter monitors and ECG data in Friedreich ataxia patients

- We want to learn about the full range of cardiac dysfunction that can occur in Friedreich Ataxia

- We are interested in studying the cardiac abnormalities and severities of Friedreich ataxia patients

 

What Is Involved In Participation?

 

- Talking with the study coordinator about your Friedreich Ataxia history

- Identifying where your medical records are kept

- Signing medical record release forms for us to obtain your records

- You will not need to come to the Children’s Hospital of Philadelphia in order to participate

- This is a research study and no personal results will be reported back to you directly

- Your information will be kept strictly confidential

- Your participation is entirely voluntary and the study team will be available to answer questions about your rights as a research subject

 

For More Information Please Contact:

Erin Paulsen, Study Coordinator

(267) 426-9738

This email address is being protected from spambots. You need JavaScript enabled to view it.  

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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