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The Sleepy Road to Diagnosis and Deprivation: A Narcolepsy Patient Story, Part 1

The Sleepy Road to Diagnosis and Deprivation: A Narcolepsy Patient Story, Part 1

I want to start off by saying that the intention behind my words are not to convey or express any sort of anger, hatred, or resentment. I am choosing to…

Continue Reading The Sleepy Road to Diagnosis and Deprivation: A Narcolepsy Patient Story, Part 1
Could This Experimental Drug Treat Extreme Hunger in Prader-Willi Syndrome?

Could This Experimental Drug Treat Extreme Hunger in Prader-Willi Syndrome?

According to a story from labblog.uofmhealth.org, one of the defining features of Prader-Willi syndrome, a rare disorder, is hyperphagia, which is extreme overeating and obsession with food. While this is…

Continue Reading Could This Experimental Drug Treat Extreme Hunger in Prader-Willi Syndrome?
A New Clinical Trial is Underway for Pancreatic Cancer

A New Clinical Trial is Underway for Pancreatic Cancer

The Study: MRx0518-I-003 MRx0518-I-003 is a trial investigating MRx0518 as part of a combination treatment for pancreatic cancer, used with preoperative radiotherapy. The trial will evaluate the therapies safety and…

Continue Reading A New Clinical Trial is Underway for Pancreatic Cancer
Huntington’s Disease Patients Place Hopes in Clinical Trial

Huntington’s Disease Patients Place Hopes in Clinical Trial

According to a story from the University of Aberdeen, 57 year old Sandy Patience of Inverness, Scotland, is one of a total of 801 Huntington's disease patients from around the…

Continue Reading Huntington’s Disease Patients Place Hopes in Clinical Trial
HCU isn’t an allergy: Jessica’s Homocystinuria Story

HCU isn’t an allergy: Jessica’s Homocystinuria Story

When Jessica Karnes was a kid, she was told she was no longer allowed to eat chicken nuggets. As a self-proclaimed lifelong picky eater—and a four-and-a-half-year-old at the time—this was a…

Continue Reading HCU isn’t an allergy: Jessica’s Homocystinuria Story
Experimental Drug Proves Superior to Eculizumab as a Treatment for Paroxysmal Nocturnal Hemoglobinuria

Experimental Drug Proves Superior to Eculizumab as a Treatment for Paroxysmal Nocturnal Hemoglobinuria

According to a story from BioSpace, the biopharmaceutical company Apellis Pharmaceuticals, Inc., has recently announced the results of its phase 3 clinical trial, which was testing the company's investigational drug…

Continue Reading Experimental Drug Proves Superior to Eculizumab as a Treatment for Paroxysmal Nocturnal Hemoglobinuria
New Drug Aims to Treat Hyperphagia in Those with Prader-Willi Syndrome

New Drug Aims to Treat Hyperphagia in Those with Prader-Willi Syndrome

  Hyperphagia is an extreme hunger and obsession with food, and it is a symptom of Prader-Willi syndrome. While many of the other effects of this condition have treatments, this…

Continue Reading New Drug Aims to Treat Hyperphagia in Those with Prader-Willi Syndrome
Investigative Therapy Puts Seven Olmsted Syndrome Patients in Remission

Investigative Therapy Puts Seven Olmsted Syndrome Patients in Remission

Olmsted syndrome is a rare disease most frequently caused by mutations in TRPV3. For some patients, it is caused by MBTPS2 mutations. It is characterized by palmoplantar keratoderma (PPK) as…

Continue Reading Investigative Therapy Puts Seven Olmsted Syndrome Patients in Remission
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