Oct 162016
 
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New study results from Austrian biotech AFFiRiS support continued development of its vaccine against the key Parkinson’s protein alpha-synuclein. Today the company announced its “boost” follow-up study— funded with a $1.04 million grant from The Michael J. Fox Foundation — showed that an additional dose is safe and can elicit antibodies against alpha-synuclein.

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Author: Maggie McGuire Kuhl Publication: Fox Feed blog  Date: 09-07-2016

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Sep 212016
 
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We are excited to announce the approval of Eteplirsen (now renamed Exondys 51), the first drug that treats the underlying cause of Duchenne…
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DV Note: September, 2016 the FDA gave contingent approval for the drug Exondys 51 to treat people with Duchenne Muscular Dystrophy (DMD) exon 51 abnormality. This will treat a percentage of people with DMD, but it is hopeful the drug could be modified to treat other exon abnormalities in DMD and possibly in other genetic disorders.

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Author: Catherine Jayasuriya  Publication: Coalition Duchenne  Date: 09-19-2016

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Sep 042016
 
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“By creating healthy muscle cells from the patient’s skin cells, the scientists avoid any risk of the cells being rejected by the patient’s immune system. Unlike traditional gene therapy, the faulty gene is corrected in cells outside the body”…

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Author: Lussy Publication:  Healthline.Website  Date: 08-28-2016

Jul 222016
 
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New York’s first medical marijuana dispensary opened in January, over a year after the state became the 23rd to legalize the drug for medical use and the fifth to approve Parkinson’s disease (PD) as a condition for its use. This news understandably led the patient and physician communities to ask a number of research and clinical questions about marijuana and Parkinson’s.

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Author: Rachel Dolhun, MD Publication: Michael J Fox Foundation Foxfeed Date: 02-01-2016 

May 242016
 
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Motor fluctuations are “off” times, when medication — namely levodopa (the “gold standard” treatment for Parkinson’s) — is not working optimally and Parkinson’s symptoms …

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Author: Rachel Dolhun, MD Publication: The Michael J Fox Foundation FOXFEED Blog Date: 05-23-2016

 

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Apr 282016
 
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On Monday, April 25 the Muscular Dystrophy Association (MDA) along with MDA clinical experts testified before an FDA advisory committee. They express optimism for this new drug, eteplirsen, helping people with Duchenne Muscular Dystrophy (DMD) and it opening doors for treating more genetic disorders. Eteplirsen is an Exxon shipping drug which is designed to skip certain parts of defective genes to allow the genes to still express a protein. In DMD this drug therapy would basically turn DMD into a mild form of Backers Muscular Dystrophy. Backers Muscular Dystrophy is already much more mild than Duchenne Muscular Dystrophy. This treatment would help thousands of people live a healthier and longer life.
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Publication: MDA Outreach Date: 04-25-2016

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Jan 072016
 
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Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice.

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ublication: Sourced Publication:UT Southwestern Medical Center Date: 1-4-16

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Jan 022016
 
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Brooklyn Brumfield was 16 when she was given a grim prognosis.

She would likely spend the rest of her life in treatment and would need physical therapy.

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Author: Nora Olabi Publication: msn.com Sourced Publication: Houston Chronicle

Date: 12-25-15

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