From Healthy to Zebra to Unicorn

• Post author:Annie-Danielle Grenier
• Post published:November 9, 2021
• Post category:Addison’s Disease/Autoimmune polyglandular syndrome type II/Corneal ectasia

My story starts with me being a normal, healthy kid. Or was I? My parents would’ve said so, and so would I, I think. I just wasn’t very strong or…

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JIA Flares Increase with COVID-19 Lockdown

• Post author:Alyssa Stevens
• Post published:November 9, 2021
• Post category:COVID-19/Juvenile idiopathic arthritis

According to a recent article, children with juvenile idiopathic arthritis experienced higher rates of joint inflammation during the COVID-19 lockdown. Juvenile Idiopathic Arthritis (JIA) Juvenile idiopathic arthritis (JIA) proves that…

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Studies Show a Rare Disease Diagnosis Comes with Tremendously High Health Care Costs

• Post author:Alyssa Stevens
• Post published:November 9, 2021
• Post category:Rare Disease

According to a recent article, patients diagnosed with a rare disease are faced with incredibly high health care costs compared to those without a rare disease. Impact of Rare Disease…

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STAR App Supports hATTR Amyloidosis Patients

• Post author:Jessica Lynn
• Post published:November 9, 2021
• Post category:Amyloidosis/Hereditary ATTR Amyloidosis

When it comes to advances in hereditary ATTR amyloidosis (hATTR amyloidosis), biopharmaceutical company Alnylam Pharmaceuticals, Inc.  ("Alnylam") has been on top of its game. Alnylam helped develop Onpattro and is…

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Periodic Paralysis Conference 2021: An Opportunity for Community and Awareness

• Post author:James Moore
• Post published:November 9, 2021
• Post category:Periodic paralysis

On October 30-31, 2021, the Periodic Paralysis Association (PPA) hosted the Periodic Paralysis Conference in Orlando, FL. This event was an opportunity for patients to meet one another, share experiences,…

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New Technology Found To Be Better Treatment for Hereditary Tyrosinemia Type 1 and Wilson Disease

• Post author:Trudy Horsting
• Post published:November 9, 2021
• Post category:methylmalonic acidemia/Methylmalonic Acidemia/Tyrosinemia/Wilson Disease

LogicBio Therapeutics has just presented preclinical data of their GeneRide platform. This data demonstrates that the GeneRide technology is valuable in methylmalonic acidemia (MMA), hereditary tyrosinemia type 1 (HT1), and…

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Study: Xermelo for Carcinoid Syndrome Diarrhea

• Post author:Kendall Mason
• Post published:November 9, 2021
• Post category:Carcinoid Syndrome

Data from the TELEPRO-II study of Xermelo were recently published in the Journal of Cancer Management and Research. It investigated this oral therapy as a treatment for carcinoid syndrome (CS)…

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