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
Editor’s Note: This article was shared with us by our friends at Heal Canada. To see the article in its original format, please click here.
AbbVie has announced a landmark regulatory milestone with the US FDA’s approval of a supplemental new drug application (sNDA) for the combination of Venclexta (venetoclax)
In rare disease and chronic illness communities, waiting isn’t just temporary — it can become part of everyday life.
Waiting for answers, for progress, for something to change.
It can feel like life is paused while everything around you keeps moving.
But your life is still valid in the in-between. Your pace is still enough.
If you’re living in uncertainty right now, we see you — and you’re not alone.
#RareDisease #RareDiseaseAwareness #ChronicIllness #ChronicIllnessAwareness #YouAreNotAlone #PatientWorthy
Today is International CHAMP1 Awareness Day 💙
CHAMP1-related neurodevelopmental disorder is rare, but the individuals and families impacted by it are strong, resilient, and deserving of understanding, support, and visibility.
Today we raise awareness, share knowledge, and stand with families worldwide as we work toward earlier diagnoses, better resources, and continued research.
💙 Every voice matters
💙 Awareness leads to understanding
💙 Together, we are stronger
#CHAMP1AwarenessDay #RareDiseaseAwareness #CHAMP1 #PatientWorthy
We are glad you were there ! Thank you International Rett Syndrome Foundation
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