Patient Worthy Contributor – Page 49

The Shoulder Flop

Post author:Patient Worthy Contributor
Post published:April 24, 2017
Post category:Rare Disease

This article was originally published on Luke's blog and is shared here with permission. Keeping our kids safe is every parent’s number one job. Even as an adult I associate…

Editor’s Choice: Overcoming Obstacles and Fulfilling Dreams with Rare Disease

Post author:Patient Worthy Contributor
Post published:April 21, 2017
Post category:Duchenne Muscular Dystrophy Dysautonomia Dystonia Gastroschisis POTS Rare Disease Trigeminal Neuralgia (Tic Douloureux)

Happy Friday Patient Worthians! PW Contributor Tom Seaman talks about what it's like to overcome the mental toll of your rare disease diagnosis. Another PW Contributor suffering from TN, dysautonomia and…

Progressive Shopping for Superheroes

Post author:Patient Worthy Contributor
Post published:April 21, 2017
Post category:KIF1A Mutation Rare Disease

The following blog post was written on October 4th, 2016 by a #raredad whose daughter is battling a chronic illness. With his permission, we are republishing this powerful memory here.…

When You’re Disabled, But Not Disabled Enough

Post author:Patient Worthy Contributor
Post published:April 19, 2017
Post category:Dysautonomia POTS Rare Disease Trigeminal Neuralgia (Tic Douloureux)

Ever wonder what it’s like to be so sick and disabled that you can’t live a normal life but you aren’t sick or disabled enough to actually be considered disabled?…

https://pixabay.com/en/hands-world-map-global-earth-600497/

What Are You Doing for World PI Week?

Post author:Patient Worthy Contributor
Post published:April 17, 2017
Post category:CGD Primary Immunodeficiencies SCID

April 22nd through April 29th is World PI Week, dedicated to bringing awareness to Primary Immunodeficiencies (PI). If you want to participate, World PI Week has some great information for…

Editor’s Choice: NASH Hope, Sickle Cell Symptoms & Rare Disease Research Reinforcement

Post author:Patient Worthy Contributor
Post published:April 14, 2017
Post category:Nonalcoholic steatohepatitis Rare Disease Sickle cell anemia Trigeminal Neuralgia (Tic Douloureux)

Happy Friday Patient Worthians! What do you know about sickle cell anemia? We have seven facts for you! Also, we have some hope-worthy nonalcoholic steatohepatitis (NASH) news to report. What does…

Cystic Fibrosis Research Inc. Event Just Three Weeks Away! Deadline to Register is Tomorrow

Post author:Patient Worthy Contributor
Post published:April 12, 2017
Post category:Cystic Fibrosis Rare Disease

CFRI's third annual Embrace Retreat is happening May 5th - May 7th! The Embrace Retreat is specifically geared toward mothers of children and adults battling cystic fibrosis. Just in time…

Editor’s Choice: Events on Events on Events! Help Raise Sjogren’s Awareness and BOS Awareness

Post author:Patient Worthy Contributor
Post published:April 7, 2017
Post category:Bohring-Opitz Syndrome Hemophilia A Hemophilia B Myelodysplastic syndromes Rare Disease Sjogren's Syndrome

Happy Friday Patient Worthians! April is Sjogren's Awareness Month! There was also a whole day dedicated to Bohring-Opitz Syndrome. The MDS Foundation has another upcoming event as well. Lastly, last month…

https://pixabay.com/en/breathing-lungs-windpipe-2029614/

Duchenne Muscular Dystrophy Clinical Trial of Respiratory Function

Post author:Patient Worthy Contributor
Post published:April 3, 2017
Post category:Duchenne Muscular Dystrophy Rare Disease

A clinical trial is up for those suffering from Duchenne Muscular Dystrophy. An oral tablet called Idebenone will be compared to a placebo, to see if it is safe and…

Editor’s Choice: New MS Drug Approved, Narcolepsy Realities and Building a CF Team

Post author:Patient Worthy Contributor
Post published:March 31, 2017
Post category:Cystic Fibrosis Dystonia Multiple Sclerosis Narcolepsy Rare Disease

Welcome back Patient Worthians! This week we have some exciting news for the MS community. Additionally, we have some great tips on how to build your CF dream team. Also,…

Cystic Fibrosis Study Shows Significant Improvement in Lung Function

Post author:Patient Worthy Contributor
Post published:March 30, 2017
Post category:Cystic Fibrosis

Vertex Pharmaceuticals announced that two phase 3 studies involving a combination of tezacaftor / ivacaftor improved the lung function in cystic fibrosis patients that are 12 and older, with the F508del…

Ocrevus for Multiple Sclerosis Approved by FDA

Post author:Patient Worthy Contributor
Post published:March 29, 2017
Post category:Multiple Sclerosis

In two weeks, Ocrevus will be available to those suffering from both relapsing-remitting Multiple Sclerosis (MS), the most common form, and primary progressive MS, which affects about 10 to 15…

Source: Pixabay

The 3rd Edition of the WAPO ‘Global Pituitary Voice’

Post author:Patient Worthy Contributor
Post published:March 28, 2017
Post category:Acromegaly Cushing Disease Rare Disease

The World Alliance of Pituitary Organizations unites the pituitary patient community and as one of our partners, they sent us their latest newsletter! In it, you can read the chairperson's report,…

Editor’s Choice: Consequences of Unintentional Exploitation in Rare Disease

Post author:Patient Worthy Contributor
Post published:March 24, 2017
Post category:Gastroschisis Narcolepsy Pemphigus and pemphigoid Rare Disease

Happy Spring Patient Worthians! This week we have pieces from two rare women, one battling the rare disease pemphigus vulgaris, part of the pemphigus and pemphigoid family. The other is…

https://pixabay.com/en/map-usa-map-usa-united-states-1149538/

Spring is for Setting World Records and Raising ALD Awareness

Post author:Patient Worthy Contributor
Post published:March 22, 2017
Post category:Adrenoleukodystrophy Rare Disease

This week is a small win for adrenoleukodystrophy (ALD) awareness and for these mini-bikers. Kevin Bean're and Tom Ford are mini-biking across the country, from Florida to California, to raise awareness…

Iva is a patient advocate for pemphigus and pemphigoid.

A Pemphigus Vulgaris Advocate’s Plea

Post author:Patient Worthy Contributor
Post published:March 20, 2017
Post category:Pemphigus and pemphigoid Rare Disease

Iva Rauh is a Pemphigus Vulgaris patient and advocate living in Maryland. Originally from Eastern Europe where the gene for this disease is prevalent, this is her plea to the…

Editor’s Choice: Suddenly Sleepy and Beautifully Broken

Post author:Patient Worthy Contributor
Post published:March 17, 2017
Post category:Dysautonomia Hemophilia A Hemophilia B Narcolepsy Parkinson's Disease POTS Rare Disease

Welcome back to Editor's Choice! This week we have an interesting take on Parkinson's treatment. We also have an empowering article from one of PW's rare disease contributors. We are all feeling the…

https://pixabay.com/en/cup-victory-winner-award-gold-1010918/

2017 NORD Rare Impact Awards Honorees Announced

Post author:Patient Worthy Contributor
Post published:March 17, 2017
Post category:Rare Disease

The Rare Impact Awards for 2017 will be held this year, on May 18th, at 6:30 pm in Washington, DC. The Rare Impact Awards is the National Organization for Rare Disorders (NORDS)…

https://pixabay.com/en/washington-dc-c-city-cities-urban-1607746/

Is NIH Funding in Trouble?

Post author:Patient Worthy Contributor
Post published:March 16, 2017
Post category:Dysautonomia POTS Rare Disease

After lobbying during Rare Disease Week on Capitol Hill a couple of weeks ago, I was convinced we were pretty successful with our congresspersons in showing them how vital NIH…

https://pixabay.com/en/blur-bronze-coins-color-currency-1851466/

DMD Drug Sold After Pricing Problems for Pharma Company

Post author:Patient Worthy Contributor
Post published:March 16, 2017
Post category:Duchenne Muscular Dystrophy Rare Disease

I recently covered an author who had some solid advice for drug makers in regards to the rare disease pricing problem. The following drug maker may or may not have…

https://pixabay.com/en/bus-stop-sign-yellow-schoolbus-1098970/

Are You Unknowingly Exploiting Kids with Rare Disease?

Post author:Patient Worthy Contributor
Post published:March 16, 2017
Post category:Rare Disease

Imagine, three to 10 months with your new born baby in a Neonatal Intensive Care Unit (NICU) born with a potentially mortal disease or defect. You’ve battled through surgeries, recoveries,…

https://pixabay.com/en/chain-metal-chain-link-257490/

How is Friedreich’s Ataxia Linked to Inflammatory Bowel Disease and Growth Hormone Deficiency?

Post author:Patient Worthy Contributor
Post published:March 15, 2017
Post category:Friedreich ataxia

Friedreich's ataxia (FA) is a rare and painful disease. With few treatments available to treat the genetic mutation, most therapy is aimed at controlling symptoms. Clearly, these short-term band-aids aren't…

https://pixabay.com/en/malta-harbor-port-fort-wall-1964700/

Malta Boy with ROHHAD Runs for His Life

Post author:Patient Worthy Contributor
Post published:March 14, 2017
Post category:Rare Disease ROHHAD

Chucky Bartolo of Lovin' Malta introduced Jake Vella to the world yesterday. Jake suffers from the rare disease ROHHAD. While he was a regular kid for the first five years of his…

https://pixabay.com/en/united-states-capitol-politics-1675539/

A Dangerous Consequence of the 21st Century Cures Act

Post author:Patient Worthy Contributor
Post published:March 14, 2017
Post category:Primary Immunodeficiencies

We are huge proponents of the 21st Century Cures Act, as it does so much for the rare community. However, there has been an unintended consequence of it's 2016 passing…

Pixabay

Invisible No Longer, But Beautifully Broken

Post author:Patient Worthy Contributor
Post published:March 13, 2017
Post category:Dysautonomia POTS Rare Disease

Irrelevant, insignificant, ignored, un-empowered, but worst of all INVISIBLE. Let me let you in on a big secret. Those of us who are fighting everyday with the simplest of tasks,…

The Shoulder Flop

Editor’s Choice: Overcoming Obstacles and Fulfilling Dreams with Rare Disease

Progressive Shopping for Superheroes

When You’re Disabled, But Not Disabled Enough

What Are You Doing for World PI Week?

Editor’s Choice: NASH Hope, Sickle Cell Symptoms & Rare Disease Research Reinforcement

Cystic Fibrosis Research Inc. Event Just Three Weeks Away! Deadline to Register is Tomorrow

Editor’s Choice: Events on Events on Events! Help Raise Sjogren’s Awareness and BOS Awareness

Duchenne Muscular Dystrophy Clinical Trial of Respiratory Function

Editor’s Choice: New MS Drug Approved, Narcolepsy Realities and Building a CF Team

Cystic Fibrosis Study Shows Significant Improvement in Lung Function

Ocrevus for Multiple Sclerosis Approved by FDA

Editor’s Choice: Consequences of Unintentional Exploitation in Rare Disease

Spring is for Setting World Records and Raising ALD Awareness

A Pemphigus Vulgaris Advocate’s Plea

Editor’s Choice: Suddenly Sleepy and Beautifully Broken

2017 NORD Rare Impact Awards Honorees Announced

Is NIH Funding in Trouble?

DMD Drug Sold After Pricing Problems for Pharma Company

Are You Unknowingly Exploiting Kids with Rare Disease?

How is Friedreich’s Ataxia Linked to Inflammatory Bowel Disease and Growth Hormone Deficiency?

Malta Boy with ROHHAD Runs for His Life

A Dangerous Consequence of the 21st Century Cures Act

Invisible No Longer, But Beautifully Broken

Let’s Work Together!

Keep Up to Date

Learn More