As reported in a recent article by Pharmaceutical Technology, Bristol Myers Squibb (BMS) has secured US Food and Drug Administration (FDA) approval for Sotyktu (deucravacitinib)
Editor’s Note: Patient Worthy is honored to share this caregiver story with you, originally written by Aisling Melton, Parent Champion at Courageous Parents Network. In
GlaxoSmithKline has achieved a significant regulatory milestone as China’s National Medical Products Administration granted priority review status to linerixibat, an investigational treatment for cholestatic pruritus
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has accepted and granted Priority Review to AstraZeneca and Daiichi Sankyo’s supplemental Biologics License
Two of Intellia Therapeutics’ clinical trials that had been on hold since October 2025 have now been released by the FDA. According to The New
REGENXBIO encountered a significant regulatory obstacle when the FDA issued a complete response letter regarding its biologics license application for RGX-121, an investigational gene therapy
As reported on RTT News, Serina Therapeutics has initiated patient dosing in its Phase 1b registrational study of SER‑252, marking a significant milestone for the
For families affected by phenylketonuria (PKU), newborn screening can mean the difference between a healthy future and lifelong complications. PKU is a rare inherited metabolic
Tardive dyskinesia represents one of medicine’s cruelest ironies: a side effect born from the very medications designed to restore psychiatric health. For generations, treating this
Astrocytoma and glioblastoma are fast-growing brain cancers that often return after surgery. Survival for astrocytoma patients is generally four to five months. Medications that activate
Vanda Pharmaceuticals has achieved a significant regulatory milestone with the FDA’s approval of milsaperidone (Bysanti), a newly designated atypical antipsychotic medication poised to help adults
As reported by BioPharmaDive, he Food and Drug Administration (FDA) has intensified its oversight of companies marketing compounded GLP‑1 therapies, while Intellia Therapeutics, United Therapeutics,
For people living with Lynch syndrome, the threat of cancer is a constant shadow. This inherited condition affects about one in 300 people, making them
As reported on MedicalXpress, a new study published in Molecular Psychiatry suggests that a specific circular RNA found in blood, known as CDR1as, may help
As reported on BioPharmaDive, Aardvark Therapeutics has halted dosing and enrollment in its late‑stage HERO trial for ARD‑101, a first‑in‑class treatment candidate for Prader–Willi syndrome
Editor’s Note: Patient Worthy is pleased to bring you this article, originally shared with us by our friend Julie Lanford, The Cancer Dietitian. To see
The landscape of advanced breast cancer treatment is shifting. Genentech’s filing acceptance of giredestrant, an experimental oral medication, represents a pivotal moment for thousands of
As reported in Business Wire, Vico Therapeutics has initiated patient dosing in an expanded cohort of its Phase 1/2a clinical study evaluating VO659, an antisense
This image represents Stiff Person Syndrome. A rare and often invisible neurological disease that locks the body without warning. The rigid lines show muscles that will not relax. The frozen movement shows how everyday moments can suddenly stop. What looks calm on the outside often hides fear pain and exhaustion. People with Stiff Person Syndrome live with constant uncertainty. A sound stress or gentle touch can trigger painful spasms. Many are misunderstood because you cannot always see what is happening. Awareness matters. It leads to earlier diagnosis, better care and more research. It reminds those living with this disease that they are not alone. Please take a moment to learn share and listen. Visibility can change lives. 💙 #stiffpersonsyndrome #raredisease #invisibleillness #chronicillnessawareness #PatientWorthy#SeeTheInvisible #spsawareness
March is Deep Vein Thrombosis Awareness Month. 💙 🌀
Blood clots can happen to anyone, but awareness is one of the most powerful tools we have to prevent serious complications like DVT. Long travel, surgery, pregnancy, certain medical conditions, and even prolonged sitting can increase the risk. |
Take a moment today to learn the signs, move your body regularly, and encourage someone you care about to do the same. #stoptheclot #DVTAwareness #PatientWorthy
During Rare Disease Week in DC, we had the pleasure of meeting DaNice D Marshall, one of ten 2025 RareArtist Awardee winners! A telecommunications technician by trade, DaNice never painted before her granulomatosis with polyangiitis (GPA) diagnosis. She was initially hospitalized for 28 days, and no one could figure out what was wrong.
"I was told to make out a will and put my house in order. So I went home and I said, 'I don't know how to die. I know how to live.' I would lay my head down, go to sleep, and then wake up and BOOM, I'm still here. So for me, painting was salve, art is salve. It’s healing. My body is always in pain. I started with abstract art because I didn't know what I was doing, but after a while I started to do figurative art. Three years after I was diagnosed, someone noticed my art and suggested I put it out on social media. That's what I did, but really just for my family, and it took off! Since then, I've had my work on billboards in Boston. I've had nine solo shows. I've had 55 group exhibitions, was awarded a MassMOCA Residency Fellowship and several grants. All of this since I started doing figurative art in 2020.
Recently, I’ve started to convert my artwork to tactile cards and textural art, because I want to make my art accessible to neurodiverse people and those with low vision, so they can touch my art to see it.
This particular piece titled, “In Someone Else’s Shoes” depicts me, my interpretation of me. I'm always joyful. I'm always trying to bring joy, to be happy. But there's also a struggle. The shoes don't fit, I don't know where I'm going, I don't know if I'm going to make it. My disease is life threatening, so navigating the healthcare isn’t always easy. But it's what it is. I'm here, and that's all that matters. I call myself an "artivist" because I'm becoming more of an advocate for patients like me with rare diseases. I just want people to know art is society’s empathy muscle, it must be exercised daily to keep it strong ."
#RareDC2026 #RareDiseaseWeek #PatientWorthy #GPA
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