I Needed to Accept Change to Better Cope with Dystonia
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I Needed to Accept Change to Better Cope with Dystonia

In 2001, I developed severe chronic pain from a neurological movement disorder called dystonia. My life before and after dystonia are as different as night and day. I went from a very active lifestyle to…

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New Partnership Will Develop Gene Therapy for Propionic Acidemia
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New Partnership Will Develop Gene Therapy for Propionic Acidemia

According to a story from Scimex, the Children's Medical Research Institute in Sydney, Australia, has recently agreed to a new partnership with LogicBio Therapeutics, a biotechnology company from Boston, MA.…

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Potential Treatment for Niemann-Pick Disease is Granted Orphan Drug Designation in the EU
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Potential Treatment for Niemann-Pick Disease is Granted Orphan Drug Designation in the EU

According to a story from rdmag.com, the biopharmaceutical company IntraBio Inc., recently announced that its IB1000 series of investigational therapeutic compounds have been granted Orphan Medicinal Drug Designation as a…

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Finally Severe Aplastic Anemia Patients will Have Access to Eltrombopag
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Finally Severe Aplastic Anemia Patients will Have Access to Eltrombopag

Eltrombopag (Promacta) has now been approved for all individuals in the US with severe aplastic anemia. The drug will be accessible to both pediatric and adult patients. This is huge news.…

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