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 targeting mucopolysaccharidosis II (MPS II),…
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 company’s lead investigational therapy for…
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 disorder that leads to toxic…
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 involuntary movement disorder meant accepting…
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 the body’s immune system, especially…
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 battling schizophrenia and acute bipolar…
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, Merck & Co., and federal…
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 up to 80% more likely…
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 forecast how patients with major…
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 (PWS), following the discovery of…
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 article in its original…
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 women grappling with a particularly…
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 oligonucleotide (ASO) designed to target…
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. James, 73, is a partner…
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) and acalabrutinib as a first-line…
As published in Sleep Review Magazine, Axsome Therapeutics has officially begun dosing patients in its Phase 3 CLARITY trial, a study designed to evaluate solriamfetol as a potential treatment for…
Fibrosis, the accumulation of scar tissue in organs, disrupts normal organ function and leads to progressive impairment. Currently, there is no cure, and existing treatments focus primarily on symptom management…
The relationship between people with type 1 diabetes and their medication has always been complicated. For decades, managing the condition has required constant vigilance including mentally calculating food intake, predicting…
As reported on PharmaBiz, Harness Therapeutics has selected HRN001 as its lead therapeutic candidate for Huntington’s disease (HD), marking a major milestone for the company as it prepares to transition…
Click here to see Part 1 of of this 3 part pain series. If you suffer with chronic pain, you not only live with the unrelenting sensation of pain, but probably…
Patients using Zepbound for weight management now have a simpler option for administering their medication. As reported by PharmaLive.com, Eli Lilly received FDA clearance for a redesigned injection pen that…
On this episode of Wait, How Do You Spell That?, host Bree Clare sits down with Dr. Charles Link, a pioneering oncologist whose career has fundamentally shaped modern cancer medicine…
At 44 years old, I was ready to hit the reset button. In March of 2025, my partner and I stepped away from our careers. The decision wasn’t reckless, it…
Ovarian cancer is a serious gynecological cancer with no reliable screening test, resulting in limited treatment options and a low number of survivors. Each year about 3,100 Canadians receive a…
Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration has accepted for Priority Review the Biologics License Application (BLA) for garetosmab, marking a significant milestone for patients living with…
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