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
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
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
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
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
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
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
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
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
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,
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
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.
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.
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
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
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
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
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
At this year's 76th annual Liver Meeting, Novo Nordisk presented data showing that semaglutide has a positive impact on patients living with MASH (formerly known as NASH). Read more here:
patientworthy.com/2025/11/22/semaglutide-shows-liver-health-benefits-beyond-weight-loss-in-mash-p...
Today we recognize Emanuel Syndrome Awareness Day—a day to celebrate the resilience, joy, and uniqueness of children and families affected by this rare chromosome disorder. Every post, story, and shared moment helps shine a light on Emanuel Syndrome and connect the community. Together, we raise awareness and show that even rare conditions can have a powerful voice. #emanuelsyndromeawarenessday #EmanuelSyndrome #RareDisease #PatientWorthy
Hyperphagia in PWS can feel overwhelming, but there are strategies, support, and new treatments that help families manage it day-to-day. This carousel breaks down the essentials—what it is, why it happens, and the tools that actually make a difference.
You’re not alone in this journey. 💛
If you have experiences with Hyperphagia in PWS and are interested in sharing your story, click here: bit.ly/4dV7gru
#PraderWilliSyndrome #PWSAwareness #Hyperphagia #RareDiseaseCommunity #PatientWorthy
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