As reported on PharmaBiz, the US Food and Drug Administration (FDA) has approved Novo Nordisk’s higher‑dose formulation of semaglutide, Wegovy HD (7.2 mg once weekly),
I used to be really active: swimming, Tai Chi every week, practicing yoga. So, when I first felt persistent rib and back pain in late
GSK has announced a significant advancement in liver disease treatment with FDA approval of Lynavoy (linerixibat), marking the first medication specifically approved in the United
My diagnosis of rheumatic heart disease came on suddenly. I remember being 12, sitting at the doctor’s office, and was told that I have a
Researchers at Florida Atlantic University (FAU) have identified a cellular pathway that allows brain cells to share proteins and other toxic material. The study was
Lupin, a globally recognized pharmaceutical company, has achieved a significant regulatory milestone with the receipt of tentative FDA approval for Pitolisant tablets in strengths of
Editor’s Note: Patient Worthy is honored to present this article, shared with us by our friends at the Steatotic Liver Foundation (formerly the Fatty Liver
Fight. I don’t think that those who aren’t in the Huntington’s Disease community understand what the word FIGHT truly means to those of us inside
My name is Amanda. I was diagnosed with a genetic connective tissue disease called Hypermobile Ehlers-Danlos Syndrome (h-EDS) about four years ago. However, the diagnosis
My name is Faye. I’m blessed to be the wife of the love of my life, Brad, and the proud mom of two extraordinary boys
A major clinical trial has revealed disappointing results for setmelanotide, a promising obesity treatment, when used in patients carrying single genetic mutations associated with weight
As reported on PharmaBiz, Illumina and Labcorp have announced an expanded strategic collaboration aimed at accelerating precision oncology by applying next-generation sequencing (NGS) technologies more
Editor’s Note: The views, thoughts, and opinions expressed in this article belong solely to the author and do not necessarily reflect the position of Patient
Immutep Limited has announced the discontinuation of TACTI-004, a pivotal Phase III clinical trial investigating eftilagimod alfa (efti) as a first-line treatment for advanced non-small
As reported on FiercePharma, China-based biotech Dizal Pharmaceutical has reported a pivotal phase 3 success for its oral EGFR inhibitor Zegfrovy (sunvozertinib), marking a potential
Editor’s Note: The following article was originally written by Kelly Curtin-Hallinan, and shared with us by our friends at Elephants & Tea. This past St.
Arexvy Now Available for Adults Aged 18–49 with Certain Health Conditions The U.S. Food and Drug Administration has granted approval to expand GSK’s Arexvy respiratory
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
Parent Project Muscular DystrophyPPMD has a long history of working alongside our community to document treatment preferences and share those insights with regulatory authorities such as the FDA. Through town halls, listening sessions, and conversations at community events, we consistently hear that stability is a critically important outcome in both care and treatment.
PPMD has collaborated with researchers at The Ohio State University to conduct a patient preference survey that aims to better understand how individuals with #Duchenne and their caregivers value stability as an outcome of therapeutic intervention, and to capture what stability truly means in the context of treatment and care.
BOTH caregivers and individuals living with Duchenne from the same household are welcome to take the survey! Each person who wants to participate must complete the interest form in order to receive their own individualized survey link.
Check it out: osu.az1.qualtrics.com/jfe/form/SV_d5nXE3fLxqrBxsO
@OrphanDrug ConferenceFrom June 9-11 in Boston, MA, the World Orphan Drug Congress USA will once again bring together pharmaceutical companies, government officials, patient advocates, and other crucial stakeholders who contribute to developing and improving access to life-saving therapies for rare disease patients. With over 280 speakers and 2,000 attendees, this event is an opportunity to develop innovative solutions to the challenges that orphan drug development poses.
Our Chief Mission Officer, Annie Kennedy, will speak on the first day of the event at 9:20 am during the keynote titled, “Building a Patient-Centered Policy Framework for Rare Disease Drug Development.” The keynote will focus on how policymakers, regulators, and industry can collaborate to create strategies that incorporate patient insights into all stages of drug development.
You can join Annie and the EveryLife Foundation at this event for free with a guest pass. Click the following link to access the guest pass application form to secure a spot: secure.terrapinn.com/V5/sponsor-guest/11031/a0AN200000inZjFMAU?utm_source=partners&utm_medium=eve...
You can learn more about this year’s World Orphan Drug Congress USA here: www.terrapinn.com/conference/world-orphan-drug-congress-usa/index.stm?utm_source=partners&utm_med...
La Cañada Flintridge Couple Takes Chance on Baby Daughter, Wins
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Seraphina Holmes was on the brink of death and wasn’t even born yet. Multiple doctors had told her parents that she would not make it to term and said, “You
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