Rare Patient News. Well Done. - Patient Worthy

A Pilot DNA Therapy for Recurrent Respiratory Papillomatosis (RRP) Shows Promising Results

Sunniva Bean - February 19, 2020

CancerCystic FibrosisDuchenne Muscular DystrophyErythropoietic ProtoporphyriaNeuromyelitis Optica

The FDA is Developing, Approving, and Expediting Exciting New Therapies

Dravet SyndromeLennox-Gastaut syndrome

ICYMI: Positive Phase 3 Trial Results for Lennox-Gastaut Syndrome

Psoriatic ArthritisRheumatoid ArthritisSpondylarthritis

A Simple Blood Test Could Lead to Personalized Therapies for Rheumatoid Arthritis and Related Diseases

LATEST PATIENT WORTHY ARTICLES

Duchenne Muscular Dystrophy

AskFirst: A New Collaborative Program to Support Rare Disease Patients in Gene Therapy Clinical Trials

AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina. This company is dedicated to the research of gene

Myelodysplastic syndromes

Free MDS Patient & Family/Caregiver Forum in Chapel Hill, NC!

One of our partner organizations, the MDS Foundation, is hosting another free MDS Patient & Family Forum in Chapel Hill, NC. When: Saturday, March 28, 2020 Where:

Alzheimer disease type 3

Study: Results from Phases II/III of Clinical Trial of Gantenerumab, a Treatment for Alzheimer’s Disease

Roche has recently released the results of the latest phases of their study of gantenerumab, which is a treatment of a rare form of Alzheimer’s

Gaucher Disease

PR001, a Treatment for Gaucher Disease, Receives Orphan Drug Designation

Prevail Therapeutics has recently been granted the Orphan Drug designation by the FDA for their gene therapy PR001, which is intended to treat Gaucher disease.

Dystonia

How to Find Meaning and Purpose Even When You’re Suffering: Q&A with Tom Seaman, Health Coach

I recently had the pleasure of being interviewed by Sunshine Mugrabi about how I transformed my life over the past 20 years in the face

Cystinosis

AVROBIO Provides Updates on its Fabry Disease Program and Cystinosis Program

According to a story from Financial Buzz, the gene therapy company AVROBIO recently presented updated data on February 10th, 2020 at the 16th Annual WORLDSymposium being held

UPCOMING EVENTS

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A rare disease can mean a lot of time saving energ

A rare disease can mean a lot of time saving energy, and expending it quickly. We each have our own balance 🌟
Follow @spoonie_village for disease content like this! •
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#raredisease #chronicillness #chronicpain #rarediseaseday #spoonie

Living with a rare disease can reveal weakness and

Living with a rare disease can reveal weakness and prove strength at the same time. To show failure is strong, to admit vulnerability is strong. You are not weak for hurting, you are strong for dealing with it. Follow @artsyaffirmations for inspiring messages like this one 🌟 ••
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#raredisease #chronicillness #chronicpain #spoonie

Taking the weekend off can be the most productive

Taking the weekend off can be the most productive time. It’s okay if you need time and space to recuperate.
Follow @thechroniccommunity for thoughtful graphics^^ •
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#raredisease #chronicillness #chronicpain

Rare disease take a little creativity to deal with

Rare disease take a little creativity to deal with. Let that be an opportunity! •
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#raredisease #chronicpain #chronicillness

Rare diseases and mental health issues are intertw

Rare diseases and mental health issues are intertwined. It is hard to tease apart our mind and our body when our body is hurting. Rare disease needs includes a long list of doctors bills and specialists adding to the cheque book, it can be hard to squeeze in the money for your own emotional healthcare. Here are some helpful tips by @therapyforwomen how you can help yourself without paying for it. •
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#raredisease #chronicillness #chronicpain

Rare disease week is just around the corner on Cap

Rare disease week is just around the corner on Capitol Hill. On February 25th-28th, patients will learn how to advocate for their needs before meeting with the representatives who make these decisions. Will you be one of them? Read the article interviewing Julia Jenkins, executive director of EveryLife Foundation that makes it happen. She said to Patientworthy, “Without good policies that foster investment, these treatments are not going to become reality.” •
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#raredisease #chronicpain #chronicillness #rarediseaseweek #everylifefoundation

Cystic fibrosis is caused by a gene mutation that

Cystic fibrosis is caused by a gene mutation that causes too much mucus to block up the respiratory and digestive systems. The disease can be helped with treatment and healthy habits. •
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#raredisease #chronicillness #spoonie #cysticfibrosis #cfwarrior

There are over 7,000 rare diseases, often without

There are over 7,000 rare diseases, often without any or many experts. Many rare disease patients live without answers and so without proper treatment. It is still just as real of an illness.
Follow @colourblind_zebra for more posts like this 🌟
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#raredisease #chronicpain #chronicillness #spoonie #eds #endadpkd

The spoonie life can be isolating- but sometimes t

The spoonie life can be isolating- but sometimes taking care of your health means spending time with people you care about.

TRENDING NOW ON PATIENT WORTHY

Study: Pozelimab Shows Positive Results in the Treatment of Paroxysmal Nocturnal Hemoglobinuria

By Kendall Mason

Deadly Rare Kidney Cancer Has a Potential New Standard of Care

By Jessica Gladwell

New Hope on the Horizon for Cystic Fibrosis Treatment

When a Genuine Voice Boosts POTS Practical Support

By Sabina Kennedy

Aplastic Anemia: What Did Baseball Do for One Happy Little Boy?

By Alisha Stone

Her Book About Living with Chronic Pain Will Make You Cheer

By Alisha Stone

The Bottom Line? Transplant is the Only Thing That Cures IPF

By Farrah Fontaine

One Little Boy Broke My Heart Until His Family Did This

By Alisha Stone

How Do You Know If Your Kid Has Behcet’s?

By Farrah Fontaine

Recently Diagnosed w/CVID? Join the Herd! #ZebraNation

By Patient Worthy Contributor

Rare Opportunity to Rub Elbows With Biotech Head Honchos

By Alisha Stone

bullseye with dart

Local Support Group Helps Those Who Suffer from Lyme Disease

By Alisha Stone

What a Freaking Nightmare: No One is Safe From Lyme Disease!

By Alisha Stone

Patient Worthy Succeeds 1000 Post Milestone

By Patient Worthy Contributor

CDC Zika Update – “Scarier than initially thought”

By Patient Worthy Contributor

Here’s How to Protect Your Baby Against Dangerous BPD

By Alisha Stone

Meme Making Contest – WE HAVE A WINNER #PWMemeChallenge

By Patient Worthy Contributor

<img width="500" height="185" src="https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_851/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest.jpg" class="attachment-500x500 size-500x500 wp-post-image" alt="" srcset="https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_851/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest.jpg 851w, https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_150/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest-150x56.jpg 150w, https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_300/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest-300x111.jpg 300w, https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_768/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest-768x284.jpg 768w, https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_696/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest-696x258.jpg 696w" sizes="(max-width: 500px) 100vw, 500px" />

Meme Making Contest – Are YOU Worthy Enough? #PWMemeWeek

By Patient Worthy Contributor

<img width="500" height="185" src="https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_851/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest.jpg" class="attachment-500x500 size-500x500 wp-post-image" alt="" srcset="https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_851/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest.jpg 851w, https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_150/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest-150x56.jpg 150w, https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_300/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest-300x111.jpg 300w, https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_768/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest-768x284.jpg 768w, https://cdn.shortpixel.ai/client/q_glossy,ret_img,w_696/https://patientworthy.com/wp-content/uploads/2016/03/Meme-Making-Contest-696x258.jpg 696w" sizes="(max-width: 500px) 100vw, 500px" />

Meme Making Contest – Are YOU Worthy Enough? #PWMemeWeek

By Patient Worthy Contributor

Behind The Smile – Invisible Illness Awareness: Stiff Person Syndrome

By Debra Richardson

More Research is Needed for Myositis Specific Treatments

By Trudy Horsting

A New Clinical Trial is Underway for Pancreatic Cancer

By Trudy Horsting

Six Tips for Handling Body Image When Living with a Rare and/or Chronic Disease

By Trudy Horsting

‘Bosentan’ Approved by FDA to Treat Pulmonary Arterial Hypertension

By Sunniva Bean

The Power of Perspective When Living with Pain

We believe rare disease patients are people, not a diagnosis. Through education, awareness and some humor, we help patients, caregivers and support persons by providing relevant and often inspirational news and stories.

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