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
Editor’s Note: This story was originally published on Linked-In and is shared with permission by Olga Lucia Torres. In college, a clinician told me I
Enpatoran offers hope for millions suffering from lupus skin manifestations Pharmaceutical company Merck KGaA has announced a significant milestone in lupus treatment research. As reported
As reported on PharmaBiz, Vanda Pharmaceuticals has launched a new clinical trial, known as Thetis, to evaluate whether its neurokinin‑1 (NK‑1) receptor antagonist Nereus (tradipitant)
Just as we patiently nurture the things we hope will bloom, we deserve that same patience for ourselves. Healing, growth, and becoming don't happen overnight. Trust the process, honor your journey, and remember that every small step forward matters. 🌷🌼
#resilience #healingtakestime #PatientWorthy
EveryLife Foundation for Rare DiseasesReady to share your story and take part in rare disease advocacy? Join the EveryLife Foundation for Rare Diseases for a virtual session, “How to Get Involved with EveryLife,” on Thursday, June 11 at 6 PM ET. You’ll hear about our programs, events, and the many ways to engage in advocacy through the Foundation. Register now at: everylifefoundation.org/intro
Want to start connecting now? Sign up for our newsletter so you never miss an opportunity: everylifefoundation.org/newsletter-signup/
Cure Syngap1Rafael loves water, nature, trees, and listening to Duo Karma. He is active, social, and loves being around other children. A cure for SYNGAP1 would help Rafael better regulate his behavior and sensory overwhelm. It would also give him the words to say “I love you” or tell his family when something hurts.
It's Syngap Awareness Month. We're counting down to 6.21!
Want to know why 6/21? Find out here: cureSYNGAP1.org/Splash26
#SYNGAP1 #CureSYNGAP1 #SYNGAP1 #CureSYNGAP1 #SplashforSYNGAP #SYNGAP1Awareness
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