Editor's Note: This is the second part in a series of excerpts written by Dana Langston. Chapter 4: The Internal Shift We are living in the "waiting room" now. The…
Editor's Note: The following is the first part of excerpt written and submitted to us by Dana Langston. There is a specific kind of internal battle that happens when you've…
On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological…
Patients living with chronic or rare diseases and the caregivers coordinating appointments, medications, and daily life often carry a quiet question alongside the medical work: what will last beyond the…
As reported on PharmaBiz, the US Food and Drug Administration has broadened the approved use of GSK’s respiratory syncytial virus (RSV) vaccine, Arexvy, to include adults aged 18 to 49…
People with rare and chronic disease often suffer from long term pain. New ways to alleviate this pain are needed. According to a press release from Tris Pharma, the results…
“Do not go gentle into that good night. Rage, rage against the dying of the light.” --Dylan Thomas One may have thought, after six months under constant supervision in a…
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 has been a shift in…
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…
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 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…
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…
AstraZeneca and Daiichi Sankyo’s antibody‑drug conjugate Datroway (datopotamab deruxtecan) has been granted Priority Review by the U.S. Food and Drug Administration (FDA) for use as an initial treatment in adults…
After being diagnosed with a rare disease or any other chronic health condition (in my case I have dystonia) and learning it is not a temporary problem (or if you…
Elevar Therapeutics has achieved a significant regulatory milestone with the US Food and Drug Administration's acceptance of a resubmitted New Drug Application for rivoceranib in combination with camrelizumab as first-line…
Sharing My Stripes 🦓 This Rare Disease Month, I'm honored to share my journey living as someone with a rare disease and what Rare Disease Month means to me. This…
A growing body of research shows that sleep quality, dietary sodium, and hypertension are more closely linked than once believed — and two major healthcare organizations are joining forces to…
As reported by the European Pharmacy Review, a combination approach using Johnson & Johnson’s small‑molecule therapy Caplyta (lumateperone) alongside standard antidepressants continues to show promising results for adults with major…
If you got past the title without rolling your eyes or getting angry (I understand if you did either or both as you will see below), I hope you will…
The U.S. Food and Drug Administration (FDA) has approved Yartemlea (narsoplimab‑wuug), marking the first authorized therapy for hematopoietic stem cell transplant‑associated thrombotic microangiopathy (TA‑TMA) in adults and children aged 2…
Editor's Note: Patient Worthy is honored to bring you this article, originally written by Kelsey Stanczyk, and shared with us by our friends at the Courageous Parents Network. So much…
Editor's Note: Patient Worthy is proud to share this story from the Courageous Parents Network, originally written by Kelsey Stanczyk. When I first became a nurse, I worked in an…
As reported on FierceBioTech, Biohaven closed out the holiday period with disappointing news: its investigational Kv7.2/7.3 potassium‑channel modulator, BHV‑7000, did not demonstrate efficacy in a phase 2 clinical trial for…
As reported by Forbes, the U.S. Food and Drug Administration has granted approval to Flow Neuroscience for the first prescription brain stimulation device designed for home use in treating major…
As reported on Science Daily, a recent clinical study suggests that a traditional Chinese herbal remedy may offer a unique advantage in treating major depressive disorder (MDD) while paving the…
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