Editor's Note: We're honored to share this patient story-share by Jess Ippolito, originally published by our friends at Stork Genetics. To see the article in its original format, please click…
Xspray Pharma, the developer of the drug dasatinib, was recently issued a Complete Response Letter (CRL) by the FDA while waiting for corrective actions, according to Cancer Network. Based on…
Sanofi has announced encouraging results from a Phase 2 clinical trial of efdoralprin alfa, an investigational therapy for alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the liver and…
I’ve suffered from Crohn's and fibromyalgia since I was a child. I was told I had “food allergies.” My fibromyalgia was hidden from me until adulthood. In 2020, I was…
Grieving families who have lost loved ones to severe allergic reactions are calling for urgent reforms to the way deaths from anaphylaxis are investigated in England and Wales. These families,…
BridgeBio Pharma has reported decisive success in a pivotal phase 3 trial for its rare disease drug BBP-418, aimed at treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Reported by Fierce…
Sanofi has announced encouraging results from its Phase 2 ElevAATe clinical trial evaluating efdoralprin alfa (SAR447537), a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, for the treatment of emphysema caused…
Merck & Co has taken a significant step forward in its immunology pipeline by initiating Phase IIb clinical studies for tulisokibart (MK-7240), its investigational therapy targeting immune-mediated inflammatory diseases. According…
This patient story is sponsored by AVEO Pharmaceuticals, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…
Women living in areas of the United States with poor air quality, particularly neighborhoods with high motor vehicle emissions, face a greater risk of developing breast cancer, according to a…
A significant shift could soon be underway in the management of severe allergic reactions, as new research highlights intranasal (IN) adrenaline as a compelling, needle-free solution for anaphylaxis. This development,…
I’ll never forget the day I was told I have ulcerative colitis. Suddenly, I had both an answer and a thousand new questions. There are a lot of emotions that…
Denali Therapeutics (Nasdaq: DNLI) announced that the U.S. Food and Drug Administration (FDA) has extended the review period for its Biologics License Application (BLA) for tividenofusp alfa, an investigational therapy…
Portal Therapeutics, in partnership with GondolaBio, has achieved a milestone with the FDA granting orphan drug and fast track status to its oral therapy candidate, PORT-77. This investigational drug, according…
Genentech, a member of the Roche Group, has unveiled results from two pivotal Phase III trials—MEERKAT and SANDCAT—evaluating the investigational drug vamikibart for uveitic macular edema (UME), a sight-threatening complication…
A new expert consensus calls for a fundamental shift in how hormone replacement therapy (HRT) is offered to breast cancer survivors suffering severe menopausal symptoms. Published in Menopause on 30…
Written by Maria Palombini Answer: at the time of diagnosis, it should be one of the first actions you, the patient and/or caregiver, should take. Why? Receiving a diagnosis with…
In a major advance for patients with the rare eye disease keratoconus, Glaukos has secured U.S. Food and Drug Administration (FDA) approval for Epioxa, an incision-free corneal cross-linking therapy. As…
In a groundbreaking move to reduce the emotional toll and diagnostic delays associated with breast cancer detection, the University of Chicago Medicine has introduced BIOPSY NOW, a patient-centered initiative offering…
Editor's Note: Patient Worthy is proud to share this blog post by Carmen D., a cancer survivor and affectionately known as The Chemo Diva. To see the article in its…
A groundbreaking study from the MRC London Institute of Medical Sciences (LMS) is reshaping how researchers approach treatment development for rare genetic disorders. Led by Dr. André Brown and the…
On October 2, 2025, Novartis announced a significant milestone in the treatment of chronic spontaneous urticaria (CSU): the US Food and Drug Administration (FDA) approved Rhapsido® (remibrutinib) as the first…
In a significant advancement for HIV prevention, the Centers for Disease Control and Prevention (CDC) has issued a strong recommendation for Yeztugo (lenacapavir), Gilead Sciences’ long-acting, twice-yearly pre-exposure prophylaxis (PrEP)…
According to a press release from Syndax Pharmaceuticals, the developer of Revumenib, the drug met its primary endpoint resulting in complete remission (CR) or CR with partial recovery. Relapsed or…
Initial results from the TROPION-PanTumor03 phase 2 trial, presented at the 2025 ESMO Congress, show that the combination of DATROWAY® (datopotamab deruxtecan) and rilvegostomig offers significant tumor responses and disease…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
