X-linked Hypophosphatemia
How One XLH Diagnosis Sparked Conversations Across Three Generations
Editor’s Note: Patient Worthy is pleased to share this story, submitted to us by Connie B. What if one diagnosis could change the way an
Vanessa’s Acoustic Neuroma Story
Optogenetic Therapy
October 9th: PANS/PANDAS Awareness Day
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Parkinson's Disease
New Research Project Will Explore Why Parkinson’s Affects Women and Men Differently
Two-year University of Arizona study will use human brain-cell models to investigate sex-specific biology and help guide future research and clinical trials. Critical Path Institute
Rare Disease
Genetic Testing: The Future is Here
Genetic testing is now more available than ever before. For those who suspect a genetic disorder, the best route is often through a genetic counselor.
Limb-girdle muscular dystrophy
Body Bound by Earth, Mind Master of the Cosmos: My Journey with LGMD2R
My name is A. Sabesan. I am 25 years old, living in Chennai, Tamil Nadu, India. Up until the age of 18, my life was
Hemophilia A
FDA Delays Decision on Novo’s Hemophilia A Therapy Over Manufacturing Concerns
As reported on BioSpace, the FDA has extended its review of Novo’s investigational hemophilia A therapy denecimig due to manufacturing-related concerns, although the company says
Bladder cancer
Bladder Cancer May Be Getting Easier to Detect
As reported on Inside Precision Medicine, researchers at Stanford Medicine and the VA Palo Alto Health Care System have created a new urine test called
Rare Disease
Seen, Heard, and Believed: Why the Patient Voice Matters
Editor’s Note: The following story was submitted to us by Pam Mason, Patient Speaker & Advocate. If you looked at a picture of me, you
Colorectal cancer
Becky’s Journey with Metastatic Colon Cancer
I’ve survived Stage 4 colon cancer for about 10 years. I went to the doctor for a routine colonoscopy. They said I had a long
Metabolic dysfunction-associated steatohepatitis
Wegovy Linked to Normal Liver Fat Levels in Small Analysis of Adults With Obesity
As reported on Medical Daily, small exploratory analysis suggests that semaglutide, the active ingredient in Wegovy, may substantially reduce liver fat in some adults with
Limb-girdle muscular dystrophy
I Live With LGMD. I Want Patients to Help Shape What Comes Next.
When I was seven, my parents were trying to understand why I was becoming ill. I spent time in hospitals and underwent painful muscle biopsies.
Rare Disease
My Big Sister
Editor’s Note: Patient Worthy is honored to present this story, shared with us by our friends at the Courageous Parents Network. Originally written by Dr.
Rare Disease
FDA Clears First Expandable Heart Valve for Pediatric Patients
As reported on Becker’s Hospital Review, the FDA has approved a first-of-its-kind pulmonary heart valve that can be enlarged after implantation, potentially allowing the device
Cancer
Galleri Moves Closer to FDA Decision as Evidence on Multi-Cancer Blood Testing Evolves
As reported on Becker’s Hospital Review, an FDA advisory committee has backed Grail’s Galleri blood test, bringing the U.S. closer to potentially having its first
Cancer
Myth Buster: You Don’t Need a High Protein Diet
Editor’s Note: Patient Worthy is honored to share this article from our friend Julie Lanford, MPH, RD, CSO, LDN – a.k.a. The Cancer Dietitian. To
Chronic Lymphocytic Leukemia
The Weight of Thank You
Editor’s Note: Patient Worthy is honored to share the following story from our friends at Elephants & Tea, originally written by Michelle Lawrence, a patient
Dementia
Muscle Loss May Be More Closely Linked to Dementia Risk Than Body Weight, Review Finds
As reported on SciTechDaily, a large research review suggests age-related losses in muscle mass, strength, and physical function may have a stronger association with dementia
IgA Nephropathy (IgAN)
Phase 3 IMAgINATION Trial of Sefaxersen Meets Primary Endpoint in IgA Nephropathy
Investigational once-monthly therapy significantly reduced proteinuria versus placebo at 37 weeks, according to interim findings from the phase 3 study. As reported on PharmaBiz, Roche
Brain Cancer
Perpetually Healing
Editor’s Note: Patient Worthy is proud to share the following story from our friends at Elephants & Tea, originally written by Dillon Groover, a brain
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On PANDAS Awareness Day, we recognize the children and families navigating a condition that can bring unexpected and often overwhelming changes. Today is about increasing understanding, encouraging compassion and making sure those affected feel seen and supported.
#pandasawarenessday #pandasawareness #rarediseaseoftheweek #raredisease #rarediseaseawareness #shareyourstory #PatientWorthy
Charcot-Marie-Tooth Association
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OPMD Association Non-Profit for Oculopharyngeal Muscular DystrophyJoin the Global OPMD Patient Registry today! The OPMD Association has taken an important step by partnering with CoRDS (Coordination of Rare Diseases at Sanford) Research. Coordination of Rare Diseases at Sanford Research is a a nonprofit research institution and a centralized international patient registry for all rare diseases. We coordinate the advancement of research into more than 10,000 rare diseases. Here’s how:
*We work with patient advocacy groups, individuals and researchers.
*We capture health information from individuals with a rare diagnosis, undiagnosed patients, unaffected carriers or at-risk patients.
*We connect researchers and patients and notify our participants of emerging clinical trials.
*We make the registry accessible. Participants can enroll for free and researchers can access it for free.
Global OPMD Patient Registry- opmd.org/registry/
Help spread the word and increase awareness by sharing this initiative with others. 💙💛
#OPMDassociation #OPMD #OculopharyngealMuscularDystrophy #OPMDPatientRegistry
