The history of cystinosis is one of the most successful examples of rare disease research leading to meaningful treatments. In a recent interview with the
This is my son Nikolai. Nikolai was recently diagnosed with Mucopolysaccharidosis type 2, or “Hunter Syndrome.” A rare genetic form of childhood dementia, it’s caused
As reported on PharmaBiz, a combination of Imfinzi (durvalumab) and Imdelltra (tarlatamab) has delivered significant improvements in survival outcomes for patients with extensive-stage small cell
At the age of 18, I was diagnosed with a rare swallowing disorder called Achalasia. At the time of this video, I had undergone several
In honor of World Duchenne Awareness Day on September 7th, Mal Duffin has invited us to share this app designed for parents whose children are living
As reported on World Pharma News, the U.S. Food and Drug Administration has granted approval to daraxonrasib, an oral RAS-targeting therapy, for adults with metastatic
A novel investigational Ebola vaccine has shown encouraging safety and immunogenicity results in a first-in-human clinical trial, supporting further development of an alternative vaccination strategy
Editor’s Note: Patient Worthy is honored to share this article, originally written by David St. Denis, President & Director at Anteris Technologies. Transcatheter aortic valve
Although Nancy Wexler’s efforts were largely responsible for creating a genetic test to locate the Huntington’s gene, she refused to take the test herself saying
As reported on Fierce BioTech, Guardant Health has secured U.S. Food and Drug Administration approval for its Guardant360 CDx liquid biopsy test as the companion
As reported on Science Daily, researchers at the University of Michigan have identified a potential new therapeutic approach for aggressive prostate cancers that become resistant
GPRC5D-Targeting Approach Positions Company to Expand Post-BCMA Treatment Options As reported on Fierce BioTech, Bristol Myers Squibb announced positive results from its pivotal Quintessential trial
Editor’s Note: We’re honored to share the following article from our friends at the Courageous Parents Network. To see this article in its original format,
Editor’s Note: Patient Worthy is honored to share this article from Rareatives, originally written by our friend Jessica Lynn. To see the article in its
Editor’s Note: Patient Worthy is honored to share this story from our friends at Elephants & Tea, originally written by Bill Phillips, a colorectal cancer
A single genetic change can have dramatically different effects from one person to the next. That is the reality for people living with PTEN hamartoma
As reported on QZ, the U.S. Food and Drug Administration has approved a new indication for Eli Lilly’s tirzepatide (Mounjaro), allowing the therapy to be
As reported on MedCity News, the U.S. Food and Drug Administration has approved Takeda Pharmaceutical’s Mimrylo (rusfertide) for the treatment of erythrocytosis in adults with
This is TONIGHT The Obesity SocietyNurse practitioners are at the forefront of transforming obesity care—are you ready to lead the way?
Don’t miss The Obesity Society’s FREE interactive webinar, “Best Practices in Obesity Management,” designed to empower NPs with the latest evidence-based strategies for treating obesity as a complex, chronic disease.
What you’ll gain:
✨ Tools to communicate with empathy and reduce stigma
✨ Practical nutrition, physical activity, and behavioral approaches
✨ Guidance on making informed treatment decisions, including medications
✨ Tips for collaborating with multidisciplinary teams to optimize patient outcomes
🗓️ Tuesday, September 15, 2026
🕖 7–9 pm ET
Secure your spot now: ow.ly/q8sb50ZM7qJ
Let’s advance obesity care together—register today!
#ObesityMedicine #PrimaryCare #NursePractitioners #ObesityCare
From our friends Global DARE Foundation #refsumdiseaseDon’t miss the latest findings from the Global DARE Foundation's food testing program coming out this September. These bring together our work and collaboration between metabolic dietitians, researchers and the Foundation.
As we begin planning the 2027 food testing program, we'd like to hear your suggestions for individual food items that you'd like to see tested.
Please email [email protected] with your suggestions for items not already tested. Thank you for your continued support of the Refsum food testing and diet guide. It makes meal choices easier for everyone with Refsum!
#globaldarefoundation #refsumdiet
From our friends FSHD Society
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