As reported on drugs.com, Bristol Myers Squibb (BMS) has announced encouraging interim results from the Phase 3 portion of the SUCCESSOR-2 clinical trial evaluating oral
As reported on FierceBioTech, the U.S. Food and Drug Administration has released its complete response letter (CRL) detailing why it declined to approve Regenxbio’s experimental
As covered on PharmaBiz, Roche has reported topline results from the phase III persevERA Breast Cancer study, which evaluated the oral selective oestrogen receptor degrader
Editor’s Note: Patient Worthy is proud to share this article from our friends at the Courageous Parents Network. A parent often can sense when there
Daiichi Sankyo has taken a significant step forward in expanding treatment options for early-stage breast cancer patients by submitting a supplemental New Drug Application (sNDA)
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
Your future self called... 🧡
#encouragement #rarediseaseawareness #chronicdiseaseawareness #selflove
Lynavoy Achieves FDA Approval: A Breakthrough Treatment for Cholestatic Pruritus in Primary Biliary Cholangitis bit.ly/3O3SNRz Read now at PatientWorthy.com
#PatientWorthy #FDAapproval #CholestaticPruritus #PrimaryBiliaryCholangitis #PBC
For some people living with rare diseases, weight loss and weakness aren’t simply about nutrition or appetite. Underlying illnesses can change how the body uses energy and muscle, leading to significant physical decline over time. Raising awareness about these complications helps highlight the serious ways certain diseases affect the body and why comprehensive care and research are so important. #RareWordOfTheWeek #RareDisease #RareDiseaseAwareness #PatientWorthy
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
