What happens when a pharmaceutical first-mover discovers its historic achievement may not be a blockbuster? Arvinas and Pfizer found an answer this week by handing
What is Parkinson’s Disease – known as PD for short? Parkinson’s Disease was first discovered in 1817 by a British doctor named James Parkinson. From
A recent article by Dr. Rosalie Greenberg explores the possible relationship of infections caused by ticks and psychiatric conditions in children, including PANS or PANDA
Abeona Therapeutics Inc. has announced a significant expansion in access to ZEVASKYN, marking the activation of Children’s Hospital of Philadelphia (CHOP) as the sixth Qualified
As reported on PharmaBiz, Regenxbio has announced encouraging topline findings from the pivotal Phase III portion of its ongoing AFFINITY DUCHENNE clinical program evaluating RGX-202,
Biossil, based in Toronto and co-founded by Dr. Alexander Mosa (Chair, CSO and co-founder) and Anthony Mouchantaf (CEO and co-founder), is making headway for rare
As reported on PharmaTimes, Poolbeg Pharma has secured a Canadian patent protecting the use of p38 MAPK inhibitors, including its lead candidate POLB 001, for preventing
Editor’s Note: This story was originally published on Linked-In and is shared with permission by Olga Lucia Torres. She waited an hour and fifteen minutes to
As reported on Healio, emerging real-world evidence suggests that glucagon-like peptide-1 (GLP-1) receptor agonists—and related dual agonists—may play a significant role in lowering the risk
I remember the day I realized something was wrong, as clearly as if it happened this past weekend. It was a beautiful morning in June.
Systemic lupus erythematosus punishes patients twice. First comes the disease itself, an autoimmune disorder where the body attacks its own tissues, causing debilitating fatigue, joint
As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has approved Partner Therapeutics’ bispecific antibody Bizengri for adults with cholangiocarcinoma harboring an NRG1
Editor’s Note: Patient Worthy is honored to share this submission from Katie DiLorenzo, SVP, Patient and Pharmacy Services, PANTHERx® Rare. If you’re living with a
Mesenchymal stromal cells (MSCs) have tantalized the medical world for years. These versatile immune-modulating cells offer genuine hope for diseases ranging from epidermolysis bullosa, a
Patient Worthy is honored to reprint this selection from The Cancer Dietitian, by Julie Lanford MPH, RD, CSO, LDN. It’s important to know that no single
I am sharing my journey to give a voice to patients who suffer from extreme poly-pathology and are often left behind by the medical system.
For decades, colorectal cancer treatment has followed the same playbook: surgeons remove the tumor, then patients endure months of chemotherapy hoping to catch any remaining
AskBio Inc., a gene therapy company operating independently under Bayer AG, has taken a key step forward in the development of its investigational Parkinson’s disease
Learning your limits can be one of the most difficult — and most important — parts of navigating life with a chronic or rare illness. Many people spend time figuring out what triggers symptoms, how to pace themselves, and when their body is asking for rest before reaching complete exhaustion.
Over time, listening to those signals and adjusting accordingly can become an important part of managing daily life and protecting overall well-being.
#raredisease #rarediseaseawareness #chronicillness #chronicillness #chronicillnessawareness #PatientWorthy
Friday, May 29th ZOOM United Porphyrias AssociationImportant update 👀
Our Young AHP ConnectUp with Hailey Brionez has been rescheduled to:
🗓 May 29
⏰ 6 PM ET
This is a supportive space for teens and young adults living with Acute Hepatic Porphyria (AHP), including AIP, HCP, VP, and ADP, to connect, share experiences, and feel understood by others navigating similar journeys.
You are not alone 🫂
👉 Please register to receive the Zoom link:
www.porphyria.org/events/ahpyouth
We hope to see you there ✨
#porphyria #AHP #raredisease #youngadults #patientcommunity
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