As reported on BioSpace, Moderna has entered a commercialization partnership with Italy‑based Recordati to support the development of mRNA‑3927, an investigational mRNA therapy for propionic
Osteosarcoma is a diagnosis no family should ever have to face. It is the most common primary bone cancer in children and adolescents, yet treatment
A groundbreaking genetic study published in The Journal of Clinical Endocrinology & Metabolism and reported by ScienceDaily.com has revealed that obesity and high blood pressure
Twenty years is a long time to believe a cancer chapter has closed. For two decades after my radical prostatectomy, I lived with the quiet
Editor’s Note: Patient Worthy is honored to share part 1 in an ongoing 10-part series, written and shared with us by Elena Genik. When Everything
A growing body of research shows that sleep quality, dietary sodium, and hypertension are more closely linked than once believed — and two major healthcare
Vera Therapeutics announced a major milestone in drug development on January 7, 2026, when the U.S. Food and Drug Administration granted Priority Review to the
As reported by the European Pharmacy Review, a combination approach using Johnson & Johnson’s small‑molecule therapy Caplyta (lumateperone) alongside standard antidepressants continues to show promising
A newly published Phase 1/2 study in The New England Journal of Medicine, and reported on by the Manila Times, highlights promising early results for tividenofusp
A New Approach to a Persistent Problem Celcuity has reached a critical inflection point in its pursuit of a novel breast cancer treatment. The FDA’s
As reported on Healio, glucagon-like peptide-1 (GLP-1)–based therapies are rapidly reshaping the landscape of diabetes and chronic kidney disease (CKD) management. Originally developed for glycemic
Immunoglobulin A nephropathy (IgAN) remains one of the most common primary glomerular diseases worldwide, yet effective treatments are limited. A new study, recently published in
If you got past the title without rolling your eyes or getting angry (I understand if you did either or both as you will see
Breaking the Cycle of Prescription Dependency The opioid epidemic that has devastated American communities for decades may finally have a novel challenger. According to Bezinga.com,
As reported by Clinical Trials Arena, Axsome Therapeutics has begun dosing patients in its FORWARD Phase III clinical trial, a pivotal study designed to evaluate
Brussels — On February 24, 2026, the acclaimed Netflix documentary The Remarkable Life of Ibelin received the Media & Awareness Raising Award at the EURORDIS
The Three-Year Fight That Changed Everything When Giles Turner learned his postcode could determine whether he received life-saving cancer treatment, he didn’t accept it quietly.
As reported on PM Live, a new clinical trial hub focused on epidermolysis bullosa (EB) has been established at University Medical Center Groningen (UMCG) in
Raynaud's disease affects blood flow to the fingers, toes, ears, and nose - often triggered by cold temperatures or stress. During an episode, skin may turn white, blue, then red, and can feel numb, tingly, or painful. 👐
It's more than "just cold hands." For many, Raynaud's impacts daily life in ways others may not see. Let's use this month to educate, advocate, and show understanding for those living with Raynaud's. #RaynaudsAwareness #StayWarm #PatientWorthy
Kathleen and Noah have been courageously navigating life with PPA2 deficiency. Patient Worthy is honored to share their story during Rare Disease Month and shine a light on their journey! Heart of PPA2
"Hi, I’m Kathleen and I’m Noah. Noah was diagnosed with a rare mitochondrial disease called PPA2 deficiency after experiencing four cardiac arrests before the age of two. Life with a rare disease was not something we ever imagined for our family, and yet here we are. Living with a rare disease like PPA2 means learning a diagnosis most doctors have never heard of, becoming an expert out of necessity, and carrying a constant awareness that life can change in an instant.
One of the most important things I want people to understand about PPA2 deficiency is that it is devastating and under-researched. It primarily affects the heart and energy production at a cellular level, and too often the first symptom is sudden cardiac arrest. There is no cure. There are no approved treatments. Families are left navigating survival with limited guidance, which is why research, awareness, and funding are not optional—they are urgent.
My hope for the future is for funded research, earlier diagnoses, and lifesaving treatments. I hope for a world where rare disease families are met with knowledge instead of confusion, and where children diagnosed with PPA2 deficiency are given a future instead of a prognosis.
Rare Disease Month is deeply personal to me. It’s a chance to speak up for a community that is too often overlooked, to put faces and names to diagnoses that are frequently dismissed because of their rarity. Participation matters because awareness leads to research, research leads to treatment, and treatment saves lives. For us, Rare Disease Month isn’t just about recognition…it’s about survival."
#RareDiseaseMonth #RareDisease #RareButNotAlone #WeCareAboutRare #ShareYourStripes #ShareYourStory #PatientWorthy #PPA2
Did you know? Audrey Hepburn lived with and eventually passed away from pseudomyxoma peritonei, a rare abdominal cancer. This Rare Disease Month, we celebrate her memory and support those living with rare conditions. #rarediseasemonth #pseudomyxomaperitonei #PMP #audreyhepburn #raredisease
#shareyourstory with us here: bit.ly/4dV7gru
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