Rare Patient News. Well Done. - Patient Worthy

Acid Sphingomyelinase DeficiencyFabry DiseaseGaucher DiseaseMPS IMucopolysaccharidosisPompe Disease

Sanofi is Making Strides in Lysosomal Storage Disease Research

Trudy Horsting - March 27, 2020

Spinal Muscular Atrophy

Zolgensma for Spinal Muscular Atrophy Now Approved in Japan

LupusMultiple SclerosisRheumatoid Arthritis

Despite Advancements in Precision Medicine, We Also Need to Look at Disease Broadly

Rare DiseaseSjogren’s Syndrome

Chloroquine was Touted to Treat Coronavirus. Now, This Sjögren’s Syndrome Patient Can’t Find It

LATEST PATIENT WORTHY ARTICLES

Arthrogryposis Multiplex Congenita

The European Parliament is Relaunching their Network for Rare Diseases

The European Parliament has announced that they will be relaunching their “Network of Parliamentary Advocates for Rare Diseases.” Its aim is to create a new

Rare Disease

Experts Explain how Understanding Immune Responses Could Save the Lives of Those Affected by COVID-19

Researchers all over the world are hard at work trying to develop both treatments for Covid-19 and vaccines for the illness. Professors of rheumatology at

Alopecia areata

Baricitinib Granted Breakthrough Therapy Designation for the Treatment of Alopecia Areata

There are currently no FDA approved treatments for alopecia areata, a condition that sees the loss of hair all over the body. Eli Lilly and

Rare Disease

The Positives of Social Distancing: Focusing on What You CAN Do

Social distancing sucks in so many ways. I’ll be the first to admit it. You can feel isolated, alone, stuck, scared. But, there are some

Chronic Kidney Disease

Using Machine Learning and Biopsies to Study the Origins of Chronic Kidney Disease Linked to Diabetes

According to a story from Healio, chronic kidney disease and diabetes are closely related. Kidney disease is unfortunately a frequent complication of both type 1

Cancer

The Experimental Drug Infigratinib Enters Trials in Multiple Cancer Indications

According to a story from gurufocus, the biotechnology company QED Therapeutics recently announced that the first patients have been dosed in separate phase 2 and

UPCOMING EVENTS

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Sometimes with rare diseases there is recovery, ot

Sometimes with rare diseases there is recovery, other times there is just acceptance. Understanding and expressing your needs helps you reach a balance that deals with the weight of the ailment. By asking for or accepting help when you need it, you’re respecting your health needs. Be kind to yourself, your loved ones want to be there, and will be happy to be told what they can do to help.

Thank you @worrywellbeing for this thoughtful graphic 🙏 •
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#raredisease #chronicillness #chronicpain #recovery #spoonie

The next episode of our rare disease podcast Wait,

The next episode of our rare disease podcast Wait, How Do You Spell That? is up and ready to keep you company through your quarantine! In this episode, Ilana and Rebekah chat about PKU and the time that Ilana tried to NSPKU’s Diet for a Day Challenge, thinking she’d have a leg up on low-protein eating as a former-vegan— she was wildly wrong. They talk about the challenges of restrictive diets, and why misunderstandings of PKU needs can actually be really dangerous. Also, after many embarrassing stumbles, they eventually learn how to pronounce phenylketonuria. As always, link in the bio- or look for us on Apple podcasts, Google Podcasts, Stitcher, or Podbean.

It’s our second episode of our new podcast Wait,

It’s our second episode of our new podcast Wait, How Do You Spell That? We talk with Anna Laurent about Our Odyssey (a nonprofit supporting young adults with rare diseases and chronic illness), Alagille syndrome (she teaches us how to pronounce it correctly, thank you Anna), and about how hard navigating the struggles of young adulthood is- especially when you have a rare disease- and how important it is to have a support from people who get it. Heads up, this was recorded on site at Rare Disease Day at the NIH and we goofed a bit with the audio. If you’re sensitive to noise, we recommend waiting for the next episode! Link is in the bio- you can also find us on Google Podcasts, Stitcher, Apple Podcasts, and Podbean @_ourodyssey_ @algsalliance

People with invisible illnesses sometimes struggle

People with invisible illnesses sometimes struggle to have their needs understood because they don’t wear them. The uncertainty of today’s new lifestyle effects some people more than others. Some chronic illnesses may present further risk for coronavirus or patients may be unable to access doctors who help them feel better. For this we can take on an extra awareness for our loved ones right now, and make sure people are ok even if they’re not asking for it yet.

Thank you @quotesbychristie for this graphic! •
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#raredisease #coronavirus #invisibleillness #chronicillness #spoonie

@spoonie_village has created this thoughtful set o

@spoonie_village has created this thoughtful set of reminders of how our whole society is now learning health insecurity and fear. This will pass, and when it does, society will have grown a clearer health consciousness. •
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#raredisease #chronicillness #chronicpain #coronavirus #coronavírus #spoonie

Its a strange time in the world and we are all fee

Its a strange time in the world and we are all feeling it. Rare disease patients know well what uncertainty in health means, and how to adapt to the discomfort. We must do it again, but we’re all entering this new world together. •
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#raredisease #chronicillness #spoonie #coronavirus #coronavírus

Hard times can make you even more grateful. We’r

Hard times can make you even more grateful. We’re all experiencing this together, though it may touch our lives in many different ways. Thank you to everyone on the frontline.
Thank you to @revelatori for this thoughtful graphic ☀️ •
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#raredisease #chronicillness #spoonie #coronavírus #coronavirus

It’s always vital to take care of your mental an

It’s always vital to take care of your mental and physical well being, quarantine season just might mean that will need some adjustments. Some rare disease patients are experts on the matter, and we’ve put some of their lessons together. Read our tips to make your time indoors healthy and happy, the link is in our bio ☝️☝️☝️ • •
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#coronavirus #quarantine #raredisease #chronicillness #coronavírus #selfcare

Even if it’s not law, it’s already clear the t

Even if it’s not law, it’s already clear the time has come to stay in your home. You can watch a good Irish movie to celebrate St. Patrick’s!🍀 •
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#raredisease #quarantine #coronavirus #coronavírus #chronicillness

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