Huntington's disease
The Researcher Who Would Not Be Tested
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
A New App for Duchenne Muscular Dystrophy Caregivers
- September 11, 2026
FDA Authorizes Daraxonrasib for Metastatic Pancreatic Cancer After Positive Phase 3 Trial Results
Ebola Continues To Be a Global Threat
TAVR Is Reaching Younger Patients. What Happens Decades Later?
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THE LATEST FROM PATIENT WORTHY
Breast Cancer
Guardant360 CDx Approved as Companion Diagnostic for AstraZeneca’s Etcamah in ESR1-Mutated Breast Cancer
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
Rare Disease
Dual-Drug Strategy Shows Promise Against Treatment-Resistant Prostate Cancer
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
Multiple Myeloma
BMS Achieves Milestone with Next-Generation CAR-T Therapy for Multiply-Treated Myeloma Patients
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
Rare Disease
To Stand Beside Suffering
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,
Pompe Disease
Machine Learning Maps and Discovers Clinical Endpoints in Pompe Disease Using Real-World Data
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
Colorectal cancer
When the Silence Got Loud
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
PTEN Hamartoma Tumor Syndrome
New PTEN Research Could Help Predict Cancer Risk in Rare Genetic Syndrome
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
Diabetes Type 2
FDA Expands Mounjaro’s Label to Include Cardiovascular Risk Reduction in Type 2 Diabetes
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
Polycythemia vera
FDA Clears Takeda’s Mimrylo for Polycythemia Vera, Introducing a Novel Treatment Option
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
Colorectal cancer
BioNTech Ends Phase 2 Trial of Personalized mRNA Therapy in High-Risk Colorectal Cancer
As reported on Drugs, BioNTech has decided to halt development of its Phase 2 BNT122-01 study investigating autogene cevumeran (BNT122/RO7198457) in patients with resected colorectal
Gastroesophageal junction carcinoma
Phase 3 HERIZON-GEA-01 Analysis Strengthens Survival Evidence for Zanidatamab-Based Regimens in HER2-Positive Gastroesophageal Cancer
As reported on PharmaBiz, BeOne Medicines has reported positive results from the second interim analysis of the phase 3 HERIZON-GEA-01 trial, further supporting the use
Leukemia
Think Beyond Remission to Protect the Life We Saved: A Young Man’s Journey with T-cell Acute Lymphoblastic Leukemia/Lymphoma
At 22, a young person’s world should be filled with possibilities—a career to build, places to travel, friendships to make, and a future to imagine.
Essential thrombocytosis
FDA Approves PharmaEssentia’s Besremi for Essential Thrombocythaemia
As reported on Pharmaceutical Technology, the US Food and Drug Administration (FDA) has granted approval to PharmaEssentia’s Besremi (ropeginterferon alfa-2b-njft) for the treatment of adults
Multiple Sclerosis
Novartis’ Remibrutinib Cuts Relapse Rates in Phase III Multiple Sclerosis Studies
In a recent press release, Novartis has reported positive topline results from its Phase III REMODEL-1 and REMODEL-2 trials, showing that the investigational oral Bruton’s
Metastatic Breast Cancer
EU Approves Enhertu Plus Pertuzumab for First-Line HER2-Positive Metastatic Breast Cancer
As reported on Business Wire, the European Commission has approved trastuzumab deruxtecan (Enhertu) in combination with pertuzumab for the first-line treatment of adults with unresectable
Rare Disease
Novartis Suspends Eight CAR-T Studies After Patient Deaths Trigger Safety Review
As reported in the European Medical Journal, Novartis has paused eight clinical studies evaluating its experimental CAR-T cell therapy, known as rap-cell, after three trial
Neurofibromatosis Type 1
Cherry Angiomas May Represent an Underrecognised Vascular Feature of Neurofibromatosis Type 1
As reported in the European Medical Journal, new research suggests that cherry angiomas occur more frequently and at an earlier age in people with neurofibromatosis
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“The Peace of Wild Things
When despair for the world grows in me
and I wake in the night at the least sound
in fear of what my life and my children’s lives may be,
I go and lie down where the wood drake
rests in his beauty on the water, and the great heron feeds.
I come into the peace of wild things
who do not tax their lives with forethought
of grief. I come into the presence of still water.
And I feel above me the day-blind stars
waiting with their light. For a time
I rest in the grace of the world, and am free.”
― Wendell Berry, The Selected Poems of Wendell Berry
Pitt Hopkins Syndrome Mahzi Therapeutics
wefunder.com
Advancing precision therapies for rare neurodevelopmental diseases. Mahzi Therapeutics is testing the waters on Wefunder. Reserve your interest and foll...
