Experimental Treatment for Huntington’s Disease Earns Orphan Drug Designation in the EU
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Experimental Treatment for Huntington’s Disease Earns Orphan Drug Designation in the EU

According to a story from globenewswire.com, the biotechnology company Emerald Health Pharmaceuticals Inc., has announced recently that its experimental drug EHP-102 has earned Orphan Drug designation from the European Medicines…

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Interview with Dr. Michelle Krishnan: New Therapies for Neurodevelopmental Disorders
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Interview with Dr. Michelle Krishnan: New Therapies for Neurodevelopmental Disorders

Front Line Genomics has recently interviewed Dr. Michelle Krishnan, who is the Translational Medicine Leader in Rare Diseases at Roche. She focuses on rare neurodevelopmental disorders, in which she works…

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Study: AV-101 for the Treatment of Dyskinesia in those with Parkinson’s Disease
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Study: AV-101 for the Treatment of Dyskinesia in those with Parkinson’s Disease

VistaGen Therapeutics has recently been cleared by the FDA to begin the second phase of their study of AV-101, which is intended for the treatment of dyskinesia in those affected…

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Positive Data Released from Early Adult T-Cell Leukemia/Lymphoma Trial
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Positive Data Released from Early Adult T-Cell Leukemia/Lymphoma Trial

According to a story from Financial Buzz, the biopharmaceutical company miRagen Therapeutics has recently announced the release of new data from its phase I clinical study. This trial is testing…

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He Didn’t Want to Miss his Daughter’s First Birthday, so he Campaigned for a Cure for ALS
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He Didn’t Want to Miss his Daughter’s First Birthday, so he Campaigned for a Cure for ALS

As originally reported in ArcaMax,  when you get a diagnosis for a terminal rare disease, a wave of emotions crashes over life. Brian Wallach emerged clutching 'hope.' In 2017, Brian,…

Continue Reading He Didn’t Want to Miss his Daughter’s First Birthday, so he Campaigned for a Cure for ALS
Family is Determined to Make Lasting Memories with Daughter Diagnosed with Sanfilippo Syndrome
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Family is Determined to Make Lasting Memories with Daughter Diagnosed with Sanfilippo Syndrome

A family is grieving for their daughter and taking it one day at a time in the face of a terminal diagnosis.   Kelly and Andrew Mills became pregnant with…

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New Oral Drug for SMA may Help Young Children with the Most Severe Form of the Disease
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New Oral Drug for SMA may Help Young Children with the Most Severe Form of the Disease

Spinal muscular atrophy (SMA) currently has two approved treatment options. Just 3.5 years ago this number was zero and in another year or so, researchers are hoping to increase it to…

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