Rare Disease – Page 147 – Patient Worthy

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Cycle Pharmaceuticals Partners With US-Firm To Utilize 3D Printing Technology For Rare Disease Treatment Development

Post author:Meagan Fulps
Post published:January 10, 2018
Post category:Rare Disease Tyrosinemia

Cycle Pharmaceuticals shook hands with US-based firm, Aprecia Pharmaceuticals as they agreed to develop and commercialize treatments for rare diseases using the advanced technology of 3D printing, recently reported Cambridge…

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Gene Therapy Makes Noise in 2017

Post author:Meagan Fulps
Post published:January 10, 2018
Post category:Cutaneous T-cell Lymphomas Rare Disease RPE65-associated Leber Congenital Amaurosis

Gene therapy has successfully made its presence known in the medical field this last year, reported ABC News. Dr. Francis Collins of the National Institutes of Health isn't afraid to…

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This New Research Could Revolutionize Treatment for Multiple Sclerosis

Post author:James Moore
Post published:January 10, 2018
Post category:Multiple Sclerosis Rare Disease

A recent study has revealed a possible mechanism for the repair of nerves damaged by multiple sclerosis. Research at the University of California has found that gene expression in certain…

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Bioprinted Liver Tissue Could Help Treat A1AD, And it Just Got Orphan Drug Status

Post author:James Moore
Post published:January 10, 2018
Post category:Alpha-1 Antitrypsin Deficiency Rare Disease

Organovo Holdings recently stated that its 3D bioprinted therapeutic liver tissue was granted orphan drug status by the Food and Drug Administration (FDA). Taylor Crouch, the company CEO, was excited…

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RARE Carousel of Possible Dreams Kicks Off

Post author:Andres Rovira
Post published:January 10, 2018
Post category:Rare Disease

The world of rare disease is seeing a ray of sunshine with a viable partnership. Two powerhouses of patient advocacy are uniting together to bring about change, awareness and cure-searching.…

This Study Highlights The Link Between Attention Control and Movement in Hemiplegia

Post author:James Moore
Post published:January 10, 2018
Post category:Hemiplegia Rare Disease

Poor mental focus may be a contributing to the paralysis that commonly occurs after a stroke, according to a new study. A person who has had a stroke, when the…

Retrophin Gift to Establish Rare Disease Network at Children’s Hospital

Post author:Patient Worthy Contributor
Post published:January 9, 2018
Post category:Rare Disease

WASHINGTON, D.C. & SAN DIEGO — Children’s National Health System and Retrophin, Inc. (NASDAQ: RTRX) are pleased to announce the creation of the Retrophin Rare Disease Network at Children’s National.…

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A Pitcher With Thoracic Outlet Syndrome is Returning to Play For The San Diego Padres

Post author:James Moore
Post published:January 9, 2018
Post category:Rare Disease Thoracic Outlet Syndrome

Baseball pitcher Tyson Ross recently signed to a Minor League contract with the San Diego Padres. In the past, Ross pitched for the Padres from 2013-2016. Now thirty years old,…

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This Company Just Crowdfunded $1.3 Million to Fight Glioblastoma

Post author:James Moore
Post published:January 9, 2018
Post category:Glioblastoma Rare Disease

Batu Biologics just raised $1.3 million in order to continue funding the development of ValloVax, an immunotherapy treatment for cancer tumors. The crowdfunding project was started in May 2017. Initially,…

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This Man is Using YouTube to Fight the Stigma On Colitis

Post author:Sarah Lau
Post published:January 9, 2018
Post category:Rare Disease ulcerative Colitis

When you are going through the daily struggles of living with a rare disease, you feel alone. It is hard to accept the support from family and friends because they…

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The Cystinosis Research Network is Offering Scholarships to Affected Students

Post author:James Moore
Post published:January 9, 2018
Post category:Cystinosis Rare Disease

The Cystinosis Research Network (CRN) is offering scholarships to prospective college students who have been impacted by the condition. The organization offers two different scholarships, one for a student that…

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Anna Doesn’t Let Friedreich Ataxia Define Her

Post author:Meagan Fulps
Post published:January 9, 2018
Post category:Friedreich ataxia Rare Disease

Anna Gordon from West Virginia doesn't let her Friedreich ataxia define her, and she encourages anyone with a rare disease to not let it define them either, reported Public Broadcasting.…

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This Woman Just Wrote 130,000 Words With One Finger

Post author:Sarah Lau
Post published:January 9, 2018
Post category:Cerebral Palsy Rare Disease

I am always incredibly inspired by the people that don’t allow their rare diseases to prevent them from doing what they love. Whether it’s been Charity Tillemann-Dick with opera or Olivia…

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Woulda, Shoulda, Coulda for Rare Disease Families

Post author:Denise Crompton
Post published:January 8, 2018
Post category:Mucolipidosis Type III Rare Disease

Pondering “If only” and “What if “ adds anxiety for rare disease families. It seems to be a part of human nature that we do a lot of looking back,…

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Potential Fabry Disease Treatment is Granted Orphan Drug Status

Post author:James Moore
Post published:January 8, 2018
Post category:Fabry Disease Rare Disease

The pharmaceutical company Protalix Biotherapeutics, Inc. announced that a drug currently in development called PRX-102 has been granted orphan drug status by the European Commission. The treatment is being developed…

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Gene Therapy Luxturna Will Cost $850,000 To Patients With Rare Eye Disease

Post author:Meagan Fulps
Post published:January 8, 2018
Post category:Rare Disease RPE65-associated Leber Congenital Amaurosis

The FDA passed along their nod of approval for the sought after gene therapy treatment, Luxturna, reported Engadget. This will be used to help aid those made blind by a…

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This Opera-Lover with PAH Got an Encore for Life

Post author:Sarah Lau
Post published:January 8, 2018
Post category:pulmonary arterial hypertension Rare Disease

Have you been told that you had to stop doing your passion in life because of your rare disease? This is the story of opera lover, Charity Tillemann-Dick. Her life…

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The FDA Just Fast Tracked A Potential New Treatment For Glioblastoma

Post author:James Moore
Post published:January 8, 2018
Post category:Glioblastoma Rare Disease

DelMar Pharmaceuticals announced that its flagship cancer treatment drug, called VAL-083, was given "Fast Track" status by the Food and Drug Administration. VAL-083 is a possible treatment for a type…

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Supermom Cori Salchert Fosters Terminally Ill Children

Post author:Meagan Fulps
Post published:January 8, 2018
Post category:Hypoxic Ischemic Encephalopathy Rare Disease

Cori Salchert is a hero. Recently featured on the Today Show, Cori was honored for being the ultimate supermom, taking in foster children who are sick with terminal diseases, report…

Editor’s Choice: Ringing in the 2018 with Awareness

Post author:Patient Worthy Contributor
Post published:January 5, 2018
Post category:CGD Rare Disease

Welcome back, Patient Worthians! We're starting out the new year with stories of strength and awareness. This week, we have an article about a man with CSF leak, whose life…

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A Boy’s Donated Organs Just Saved The Life of This Woman From Georgia

Post author:James Moore
Post published:January 5, 2018
Post category:Nonalcoholic steatohepatitis Rare Disease

Debora Chambless was on a steady decline. She suffers from nonalcoholic steatohepatitis, the most extreme form of non-alcoholic fatty liver disease. Debora and her husband Bill are active churchgoers, and…

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Strong Parents Care for Their Two Young Children with Rare Conditions

Post author:Meagan Fulps
Post published:January 5, 2018
Post category:Hereditary hemorrhagic telangiectasia Rare Disease

No family faces life's unexpected challenges better than the family of Bella and Liam Brya. Allison and her husband, Bill, deal with the struggles of having two children, Bella and…

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The Future is Now: An Electronic ‘Nose’ Can Detect Rare Diseases by Sniffing Poop

Post author:James Moore
Post published:January 5, 2018
Post category:pediatric ulcerative colitis Rare Disease

Human beings have always speculated about the future. Science fiction is probably one of the most prominent examples, and stories are replete with robots, lasers and flying cars. But no…

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Aspiring Physician Assistant with Keratoconus Avoids Blindness with Experimental Cross-Linking Procedure

Post author:Meagan Fulps
Post published:January 5, 2018
Post category:Keratoconus Rare Disease

In 2009 Emily Rochac Argueta realized her vision was rapidly deteriorating headed towards blindness, reported Times Union. The aspiring physician assistant was diagnosed with keratoconus, a rare eye disease that…

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35th Anniversary for NORD and The Orphan Drug Act

Post author:Meagan Fulps
Post published:January 5, 2018
Post category:Rare Disease

It's been 35-years since the passing of the Orphan Drug Act, and creation of the National Organization for Rare Disorders (NORD), reports PR Newswire. Even though these organizations recognize there…

Cycle Pharmaceuticals Partners With US-Firm To Utilize 3D Printing Technology For Rare Disease Treatment Development

Gene Therapy Makes Noise in 2017

This New Research Could Revolutionize Treatment for Multiple Sclerosis

Bioprinted Liver Tissue Could Help Treat A1AD, And it Just Got Orphan Drug Status

RARE Carousel of Possible Dreams Kicks Off

This Study Highlights The Link Between Attention Control and Movement in Hemiplegia

Retrophin Gift to Establish Rare Disease Network at Children’s Hospital

A Pitcher With Thoracic Outlet Syndrome is Returning to Play For The San Diego Padres

This Company Just Crowdfunded $1.3 Million to Fight Glioblastoma

This Man is Using YouTube to Fight the Stigma On Colitis

The Cystinosis Research Network is Offering Scholarships to Affected Students

Anna Doesn’t Let Friedreich Ataxia Define Her

This Woman Just Wrote 130,000 Words With One Finger

Woulda, Shoulda, Coulda for Rare Disease Families

Potential Fabry Disease Treatment is Granted Orphan Drug Status

Gene Therapy Luxturna Will Cost $850,000 To Patients With Rare Eye Disease

This Opera-Lover with PAH Got an Encore for Life

The FDA Just Fast Tracked A Potential New Treatment For Glioblastoma

Supermom Cori Salchert Fosters Terminally Ill Children

Editor’s Choice: Ringing in the 2018 with Awareness

A Boy’s Donated Organs Just Saved The Life of This Woman From Georgia

Strong Parents Care for Their Two Young Children with Rare Conditions

The Future is Now: An Electronic ‘Nose’ Can Detect Rare Diseases by Sniffing Poop

Aspiring Physician Assistant with Keratoconus Avoids Blindness with Experimental Cross-Linking Procedure

35th Anniversary for NORD and The Orphan Drug Act

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