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Daily Archives: December 18, 2017

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Gene Editing: A New Treatment Avenue for Duchenne Muscular Dystrophy?
LaCasadeGoethe / Pixabay

Gene Editing: A New Treatment Avenue for Duchenne Muscular Dystrophy?

  • Post author:Donald Blake
  • Post published:December 18, 2017
  • Post category:Duchenne Muscular Dystrophy/Rare Disease

Patients diagnosed with Duchenne muscular dystrophy (DMD) have always lived with a grim prognosis. Many don't make it into their 20s and face debilitating symptoms years earlier-- a dark reminder…

Continue Reading Gene Editing: A New Treatment Avenue for Duchenne Muscular Dystrophy?
How Anabelle Succeeds Despite CHARGE Syndrome Will Bring You Joy
Source: Pixabay

How Anabelle Succeeds Despite CHARGE Syndrome Will Bring You Joy

  • Post author:Samuel Sachs
  • Post published:December 18, 2017
  • Post category:CHARGE syndrome

One in 10,000 babies is affected by CHARGE Syndrome. Anabelle is one of them. According to doctors, Anabelle should be deaf and blind. She should need to be fed through…

Continue Reading How Anabelle Succeeds Despite CHARGE Syndrome Will Bring You Joy
Parents Fear Tax Cut Will Endanger Their Daughter with Angelman Syndrome
Source: Pixabay

Parents Fear Tax Cut Will Endanger Their Daughter with Angelman Syndrome

  • Post author:Andres Rovira
  • Post published:December 18, 2017
  • Post category:Angelman Syndrome/Rare Disease

Jay and Amy Granzow are among the many worried parents fearing the GOP tax plan, because of the threat it brings to orphan drug research. The Manhattan Beach couple fear…

Continue Reading Parents Fear Tax Cut Will Endanger Their Daughter with Angelman Syndrome
Handsome Grant Money Aids Rare Disease Research
Source: Pixabay

Handsome Grant Money Aids Rare Disease Research

  • Post author:Andres Rovira
  • Post published:December 18, 2017
  • Post category:Hereditary Spastic Paraparesis/Rare Disease

Robbie Edwards has a rare disease, one so rare that she's one of only 17 people in the world with it. She suffers from hereditary spastic paraplegia which represents a…

Continue Reading Handsome Grant Money Aids Rare Disease Research
How Historic BRCA Research Nearly Went Off the Rails in the 80s
Source: Pixabay

How Historic BRCA Research Nearly Went Off the Rails in the 80s

  • Post author:Chloe Easterbrook
  • Post published:December 18, 2017
  • Post category:BRCA-mutated Breast Cancer/BRCA-Mutations/Rare Disease

As a scientist, Mary-Claire King has had a remarkable career related to her study of BRCA genes. She’s received a Lasker Award and has been recognized by President Obama with…

Continue Reading How Historic BRCA Research Nearly Went Off the Rails in the 80s
What Really Causes Chronic Lung Infections in Children with Cystic Fibrosis?
karmasprenger / Pixabay

What Really Causes Chronic Lung Infections in Children with Cystic Fibrosis?

  • Post author:Minden Cantrell
  • Post published:December 18, 2017
  • Post category:Cystic Fibrosis/Rare Disease

Not all bacteria are created equal, but all play a role in sustaining--or breaking down--health. And when you're talking about children with cystic fibrosis (CF), a lung disease that impairs…

Continue Reading What Really Causes Chronic Lung Infections in Children with Cystic Fibrosis?
Losing Their Children Made Them Stronger Parents
Source: Pixabay

Losing Their Children Made Them Stronger Parents

  • Post author:Andres Rovira
  • Post published:December 18, 2017
  • Post category:Mitochondrial Disease

Allison and Ben Gauvin have been to hell and back, but they manage to keep their spirits up. The couple from Connecticut lost their two children to a rare mitochondrial…

Continue Reading Losing Their Children Made Them Stronger Parents
Wake Up with a Hot Cup of Promising Psoriasis Treatment
Source: Pixabay

Wake Up with a Hot Cup of Promising Psoriasis Treatment

  • Post author:Patient Worthy Contributor
  • Post published:December 18, 2017
  • Post category:Guttate Psoriasis/Severe Atopic Dermatitis

The internet has been trying to figure out the caffeine question for years: is it good for us? Is it bad for us? Will it stunt our growth? Will it prevent…

Continue Reading Wake Up with a Hot Cup of Promising Psoriasis Treatment

Featured


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Metastatic Breast Cancer: Navigating Grief


Picture of Ralph Family walking


Rethinking What It Means to Live With Acromegaly


Illustration of mentor program members


The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

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