Step Therapy Supposedly Minimizes Risk, but for Brittany it Caused Severe Complications

  The Providence Journal recently published an article by Brittany Ricci, a student at Brown University’s Alpert Medical School, detailing her year-long struggle to receive the treatment her doctor had prescribed…

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Man with Rare Disease Organizes Musical Ensemble with Other Chronic Disease Patients 

Talk about a bittersweet symphony! Published on Yale University's online paper, recent grad Jordan Plotner -- who is living with Ehlers-Danlos syndrome (EDS) -- organized a virtual musical ensemble with other…

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Pompe Disease Treatment Has Come a Long Way Since This Patient Was First Diagnosed
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Pompe Disease Treatment Has Come a Long Way Since This Patient Was First Diagnosed

According to a story from healthbeat.spectrumhealth.org, Tasha Veinbergs was 13 years old when she was first diagnosed with Pompe disease. She was visiting the doctor to treat a small cyst…

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ICYMI: New Treatment for Scleroderma Lung Disease Approved by the FDA
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ICYMI: New Treatment for Scleroderma Lung Disease Approved by the FDA

According to a story from American Nurse Today, the US Food and Drug Administration (FDA) approved a treatment on September 6th, 2019 for interstitial lung disease associated with scleroderma, a…

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UK’s NICE Recommends Coverage for Hereditary Angioedema Drug Takhzyro
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UK’s NICE Recommends Coverage for Hereditary Angioedema Drug Takhzyro

According to a story from Angioedema News, the UK's National Institute for Health and Clinical Excellence (NICE) has officially recommended the drug lanadelumab (marketed as Takhzyro) for coverage under the…

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Chief Foreign War Correspondent at NBC News Gives Account of His 4-Year-Old Son With Rett Syndrome

  Richard Engel’s son Henry, age 4, has been diagnosed with Rett syndrome, a thus far incurable genetic neurological disease. Engel is chief foreign war correspondent at NBC and has…

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Study to Investigate the Development of Lung Disease in Connective Tissue Disorders
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Study to Investigate the Development of Lung Disease in Connective Tissue Disorders

A study record from clinicaltrials.gov is investigating the appearance of lung problems in patients with disorders affecting connective tissue. While prior research has documented the appearance of interstitial lung disease…

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Study Finds The Drug Arava May be Useful for Juvenile Idiopathic Arthritis
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Study Finds The Drug Arava May be Useful for Juvenile Idiopathic Arthritis

According to a story from Juvenile Arthritis News, a recent study has found that the drug leflunomide (marketed as Arava) may be a viable treatment for juvenile idiopathic arthritis patients,…

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Results From a Study of Gene Therapy for Cerebral Adrenoleukodystrophy Appear Positive
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Results From a Study of Gene Therapy for Cerebral Adrenoleukodystrophy Appear Positive

According to a story from PharmaTimes, the gene therapy company Bluebird bio has released updated results from a clinical study of its Lenti-D gene therapy as a treatment for patients…

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Company Gears Up to Test a Gene Therapy for Phenylketonuria
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Company Gears Up to Test a Gene Therapy for Phenylketonuria

According to a story from BioSpace, the biotechnology company BioMarin Pharmaceutical Inc. has recently announced its submission of a clinical trial application with the UK's Medicines and Healthcare Products Regulatory…

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Variants of the ACE Gene Not Responsible for Disparities in Pompe Disease Treatment Impact
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Variants of the ACE Gene Not Responsible for Disparities in Pompe Disease Treatment Impact

According to a story from Pompe Disease News, a recent study has concluded the polymorphisms of the ACE gene (which are genetic variations that occur naturally) do not have an…

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Fatty Liver Disease: When You’re Not Consuming Too Much Alcohol, You’re Producing It
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Fatty Liver Disease: When You’re Not Consuming Too Much Alcohol, You’re Producing It

It's common knowledge that your Corona is doing your liver no favours. Heavy alcohol consumption isn’t great for your health. Reported in Cell Metabolism, new research gets more specific: the…

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Earlier Referral to Specialists Can Speed Up Pompe Disease Diagnosis, Study Finds
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Earlier Referral to Specialists Can Speed Up Pompe Disease Diagnosis, Study Finds

According to a story from Pompe Disease News, a recent study has found that delays in diagnosis are closely connected to whether someone displaying symptoms of Pompe disease is referred…

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Initial Trial Data Looks Promising for a Potential Treatment for Rare Epilepsies
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Initial Trial Data Looks Promising for a Potential Treatment for Rare Epilepsies

According to a story from sectorpublishingintelligence.co.uk, the drug company Ovid Therapeutics Inc. has recently announced that the early data from its phase 2 clinical trial is mostly encouraging. This clinical…

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Watch: Pharma Company Produces Cartoon Series for PH1 Awareness among Kids 

Alnylam Pharmaceuticals has developed and produced a cartoon series aimed at educating people -- especially young people -- about the pediatric rare disease primary hyperoxaluria type 1 (PH1). The campaign…

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