The U.S. Food and Drug Administration (FDA) has granted an expanded indication for Vonvendi, Takeda’s recombinant von Willebrand factor (rVWF) therapy, marking a significant advancement in the management of rare…
Stanford University has recently secured a prestigious grant from the U.S. National Institutes of Health (NIH) to accelerate the clinical development of Tiakis Biotech’s novel therapy, Elafin (also called Tiprelestat),…
In a recent study, deficiency in Vitamin D was associated with an elevated risk of developing Alzheimer’s disease. Yet there were no indications that it is the direct cause of…
Pfizer has announced breakthrough Phase 3 results for HYMPAVZI™ (marstacimab), a once-weekly subcutaneous therapy that could redefine care for people with hemophilia A or B who have developed inhibitors, as…
Editor's Note: Patient Worthy is honored to share this article from our friends at Courageous Parents Network. Parents of well children regularly receive anticipatory guidance–proactive communication about what’s coming in…
Editor's Note: This article is an opinion piece shared with Patient Worthy from our partner network. Opinion pieces may not reflect the views of Patient Worthy, its employees, or any…
Editor's Note: Patient Worthy is proud to share this caregiver story from our friends at Courageous Parents. — A parent’s perspective by Maria Hopfgarten, Courageous Parent Network Parent Champion. When…
Gene therapy for hemophilia B has reached a significant milestone: more than a decade after a single treatment, patients continue to experience stable benefits with minimal safety concerns, according to…
For families affected by rare genetic skin diseases, hope once meant daily bandages and vigilant wound care. That began to change in 2023, when US regulators approved the first-ever gene…
In our third installment from our series of reports provided by our partner TREND Community, we are bringing you several reports that analyze the effects of sleep disorders. This week…
Ireland has unveiled a new national strategy aimed at transforming the diagnosis and care of the estimated 300,000 people in the country living with rare diseases. According to The Irish…
Families and researchers in North Carolina are making urgent appeals to the FDA, highlighting the desperate need for faster approval of drugs for rare and ultra-rare diseases that threaten children’s…
After cancer treatment, many patients are left with a haunting uncertainty: Is it really gone—and will it stay that way? Even when scans appear clear, questions linger. Now, a newer…
Eli Lilly’s breast cancer drug Verzenio (abemaciclib) has achieved a significant milestone, with new study results showing it helps people with a common form of early-stage breast cancer live longer.…
A new RNA-based drug from Regeneron Pharmaceuticals and Alnylam Pharmaceuticals has shown positive results in a late-stage clinical trial for generalized myasthenia gravis (gMG), a rare, chronic autoimmune disease that…
A recent Phase I/II clinical study led by UMass Chan Medical School has shown encouraging results for a gene therapy targeting GM2 gangliosidosis, a group of rare and fatal neurodegenerative…
A groundbreaking study from the Weizmann Institute of Science and Sheba Medical Center has brought renewed hope for children with kaposiform lymphangiomatosis (KLA), a rare and life-threatening genetic disorder of…
After the death of six patients, the FDA has announced that it will require earlier and more frequent monitoring of the monoclonal antibody Leqembi, the brand name for the Alzheimer’s…
South Africa is on the brink of a major shift in HIV prevention, with plans to introduce lenacapavir—a twice-yearly injectable—at select public clinics by April 2026. As reported by All…
August 2025 was a landmark month for rare disease innovation, as the U.S. Food and Drug Administration (FDA) granted four historic approvals, each representing a first for their respective conditions,…
Researchers at UC Berkeley, DTU, and OptoCeutics in Denmark have created a non-medical light-based technology that improved cognitive function for Alzheimer’s patients who have participated in previous clinical trials. The…
HIV-associated facial lipodystrophy, a frequent complication of long-term antiretroviral therapy (ART), is characterized by abnormal fat distribution in the face and neck. This condition, often presenting as midfacial hollowing and…
The landscape of HIV treatment continues to evolve, and a significant milestone has just been reached with the FDA’s approval of Rukobia (fostemsavir), a novel therapy from ViiV Healthcare. Reported…
Acknowledgment: Patient Worthy is honored to share this story, provided to us by our friends at The AAMDS International Foundation. To see the article in its original format, please click…
When you become a parent, your life changes in an instant. You’re no longer just living for yourself—you’re living for your child. A fierce and unwavering instinct takes over, a…
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