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
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
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
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
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
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
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
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
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
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
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
A recent report from Yahoo News highlights the emergence of Guillain-Barré Syndrome (GBS), a rare but serious paralytic disorder, as a growing concern within the
Baylor Genetics is set to showcase groundbreaking innovations in RNA sequencing for rare disease diagnosis at the 2025 Advances in Genome Biology and Technology (AGBT)
In our second installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition
The U.S. Food and Drug Administration (FDA) is reviewing the first medication specifically developed for Barth syndrome, a rare and life-threatening genetic disorder. As reported
A recent report from NDTV World brings attention to a rare but significant public health event: an American man has tested positive for plague, the
Acknowledgement: Patient Worthy is proud to share this article from our friends at the Fatty Liver Foundation. As a patient, what I really want to
Health authorities in Queensland, Australia, have issued a public warning after the discovery of Naegleria fowleri—a rare but deadly “brain-eating” amoeba—in a town’s water supply.
The 1st Tuesday of the Month is a Zoom Meeting with Dr. Fink on
PLS and HSP
Everyone is welcome to Join the Zoom Meeting.
Dr. Fink will discuss HSP/PLS and answer your questions.
Here are the upcoming dates:
Tuesday, October 7th
Tuesday, November 4th
Tuesday, December 2nd
From our Friends @PBCerrs Don't miss this webinar #Oct15th
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On 9/30, we recognize Rare Cancer Day as a reminder that while each rare cancer affects few, together they impact millions. Today we raise awareness for more research and support. No one should fight alone!
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