FDA Ruling on Stem Cells

The US District Court has ruled in favor of the FDA who claimed that the use of a patient’s own stem cells to regulate therapy should be subject to the same regulations as drug therapies.

 

A company called Regenerative Sciences takes stem cells from bone marrow, processes them, and injects them back into the patient in order to treat joint pain. However, the FDA called this procedure “unapproved biological drug product manufacturing” in 2010 and ordered the prevention of the treatment.

Patients who are already genetically diagnosed with other non-dysferlin forms of LGMD, HIBM, Pompe, Bethlem, EDMD, FSHD, DMD/Becker

If you have a genetic diagnosis for one of the following muscular dystrophies, we would encourage you to join the appropriate disease specific registry. 

 

When you register with a disease specific registry, you may have access to the following services, depending on the registry:

Patients who are already genetically diagnosed with mutations in the dysferlin gene (i.e. LGMD2B/Miyoshi/Dysferlinopathy)

The international patient registry is curated for individuals who have been genetically diagnosed with dysferlinopathy (LGMD2B and Miyoshi Myopathy) through the identification of mutation(s) in the dysfelrin gene. If you have already been genetically diagnosed with mutation(s) in the dysferlin gene, please email us at patients@jain-foundation.org and we will give an overview of the community and discuss your placement in the registry.

 

Undiagnosed Patients in Other Countries

We are in communication with patients from around the world who believe they have LGMD2B/Miyoshi Myopathy. Please reach us to be placed on the international lists for diagnsotic guidance. As diagnostic support becomes more readily available, and if new programs begin in support of international genetic diagnostics, we will then be able to reach you about it. If you are interested in being listed, please send a message to: 

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