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Lafora disease

Chelsea’s Hope: The Fight Against Lafora Disease

Do you know about Lafora Disease? This rare genetic disease begins during adolescence. Your child starts their life healthy and whole, but then they decline,

Rare Disease

Steps We Can Make Every Day to Decrease Pain and Suffering

We all know that changing habits of behavior changes our life experiences, but putting this to practice is where a lot of us get stuck.

Gene Therapies

Gene Therapies for Rare Diseases: How Accelerated Approval Is Shaping the Future

An article titled “Accelerated Approval as the New Norm in Gene Therapy for Rare Diseases” from DIA Global Forum digital magazine’s May 2025 issue explores

Prader-Willi Syndrome

Resuming VYKAT™ XR Treatment Shows Significant Benefits for Prader-Willi Syndrome Patients, New Data Reveals

Soleno Therapeutics recently announced new findings regarding its investigational drug VYKAT™ XR (diazoxide choline extended-release tablets) for the treatment of Prader-Willi syndrome (PWS), as detailed

Multiple Sclerosis

Jeri’s MS Story: How One Ramp Brought a Renewed Sense of Hope

Editor’s Note: Patient Worthy is honored to share this patient story, originally authored by Jeri Burtchell. For the last month or so I’ve been trying

Alzheimer's disease

The FDA Has Approved Blood Testing for Alzheimer’s Disease

This week, the FDA approved the first blood-based diagnostic test for Alzheimer’s disease (AD). The test, known as “Lumipulse G pTau 217/β-Amyloid 1-42 Plasma Ratio,”

Cervical Dystonia

The Different Things That Help My Dystonia The Most – In A Nutshell

I have had cervical dystonia since 2001. It was very severe for many years, but over time I have been able to gain significant control

Werdnig-Hoffmann disease

For Jaicion: A Mother’s Strength, A Son’s Light, All in the Face of SMA

My name is Elena, mother to Jaicion. In 2021, I was pregnant with Jaicion, and the doctors ran tests that they would run on any

Osteosarcoma

A Rare Disease Charity Organization is Raising the Bar for Osteosarcoma Support

Myrovlytis Trust, a UK-based charity and Patient Worthy partner, has developed a new resource for those facing osteosarcoma. This devastating cancer often occurs in childhood,

Dysautonomia

Navigating a New Life with “The Trifecta” – POTS/MCAS/EDS

My name is Sarah. I am a solo mother to a beautiful five-year-old named Ensley! I’m not quite sure where to begin, but my world

Rare Disease

An Eleven-Year-Old Girl in Rowlett, Texas is Slated to Make Medical History

In 2023, Marley Mansour of Rowlett, Texas and her family were told that Marley had an extremely rare genetic condition called NARS1 disorder. Now, Marley

Metabolic dysfunction-associated steatohepatitis

Can Semaglutide Halt or Even Reverse Fatty Liver Disease?

The results released from the Phase III ESSENCE clinical trial, and recently published by Medical Xpress and in the NEJM, found that the drug semaglutide

Charcot-Marie-Tooth disease

A Surprise Diagnosis: Raiden’s Journey with Charcot-Marie-Tooth Disease and Kidney Disease

Raiden Messerli and his family thought that he had an ordinary case of the flu. Brandi Lewis, Raiden’s mother, discussed in an interview with KCCI8

Pediatric Low-grade Glioma

Meet Gabby: The Shield-maiden With the Sweetest Hugs

Acknowledgment: This patient story is sponsored by Day One Biopharmaceuticals and is promoted through the Patient Worthy Collaborative Content program. We only publish content that

Myelodysplastic syndromes

When They Were Told His Leukemia Had Spread, They Planned a Wedding to Celebrate His Life – PART 2

This article is a continuation of a previous story, and is an excerpt from original author Isabella Cristobal’s post. To read the first part of

Rare Disease

Scientists Confirm a 1958 Theory That Has Been Continually Discarded as Impossible

Professor Ronald Breslow was a chemist at Columbia University who, in 1958, proposed the theory that vitamin B1 (thiamine) could regulate metabolism pathways in the

Rare Disease

How Laura Got Her Life Back: The Impact of Organ Donation

Let’s start at the beginning of my long journey. My name is Laura, and around fifteen years ago, I was diagnosed with type-2 diabetes and

Alzheimer's disease

Never Give Up: Closing in on Alzheimer’s Disease

Immune dysfunction and inflammation have been found to play significant roles in Alzheimer’s disease. An analysis of brain tissue in an animal model reveals a potential