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The FDA Has Awarded Regenerative Medicine Advanced Therapy Designation to an Experimental Drug for X-Linked Myotubular Myopathy

• Post author:Anna Hewitt
• Post published:August 28, 2018
• Post category:Rare Disease

The investigational drug AT132 has been awarded Regenerative Medicine Advanced Therapy designation for the treatment of X-linked Myotubular Myopathy by the United States FDA. You can read the source press…

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Two Studies Investigated the Long-Term Effects of Subthalamic Deep Brain Stimulation as a Treatment for Parkinson’s Disease

• Post author:Anna Hewitt
• Post published:August 28, 2018
• Post category:Parkinson's Disease

Two studies have looked into the long-term effects of subthalamic deep brain stimulation as a treatment for Parkinson’s disease. For more detailed information on this topic, you can find the…

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Electrical Brain Stimulation Could Effectively Treat Neurodegenerative Ataxias

• Post author:Athena Lee
• Post published:August 28, 2018
• Post category:Friedreich's Ataxia/Multiple system atrophy/Spinocerebellar Ataxia

According to Newswise, new findings indicate that electrical stimulation of the spinal cord and brain may be effective in treating symptoms of a group of rare neurological movement disorders, neurodegenerative ataxias.…

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Take a Walk for Stevens-Johnson Syndrome Awareness!

• Post author:Jean Martell
• Post published:August 28, 2018
• Post category:Stevens-Johnson Syndrome

August is Stevens-Johnson Syndrome (SJS) Awareness Month! In honor of the SJS community, Friday August 31st has been designated an awareness day. What is Stevens-Johnson Syndrome? SJS is a rare, serious…

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A Story You Can’t Make Up: How Behçet’s Disease Inspired Gary to Become a Thriller Novelist 

• Post author:Jean Martell
• Post published:August 28, 2018
• Post category:Behçet's/Rare Disease

Unknown origins of birth. Overcoming enormous personal obstacles at an early age. A mysterious medical diagnosis. Making a drastic new change in life. This could - and is! - a…

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Tikomed Has Received Permission to Carry Out a Study of Their Experimental Drug for ALS in the UK

• Post author:Anna Hewitt
• Post published:August 28, 2018
• Post category:Amyotrophic Lateral Sclerosis

The speciality pharmaceutical company Tikomed has announced that the UK Medicines and Healthcare Products Regulatory Agency has granted the company permission to go ahead with a Phase 2 clinical study.…

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How Being a Good Patient Nearly Killed Me

• Post author:Patient Worthy Contributor
• Post published:August 28, 2018
• Post category:Ehlers-Danlos Syndrome/Rare Disease

I came into the hospital about four days ago at 6 am in extreme abdominal pain. I remember writhing on the emergency room bed and breathing so frantically that my…

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The First Treatment for Neurotrophic Keratitis Was Just Approved

• Post author:James Moore
• Post published:August 28, 2018
• Post category:Rare Disease/Timely

According to a story from BioSpace, the US Food and Drug Administration (FDA) recently approved the drug Oxervate (cenegermin). This treatment is the first FDA approved therapy for the rare…

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This Gene Editing Approach Could Help Treat Genetically Linked Rare Diseases

• Post author:James Moore
• Post published:August 28, 2018
• Post category:Hurler Syndrome/Tyrosinemia

According to a story from medicalxpress.com, a team of scientists at the University of Massachusetts Medical School have successfully developed a strategy for editing genes that could be used to…

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