As reported on InsidePrecisionMedicine, a new gene therapy aimed directly at the cells responsible for myelination is showing unprecedented promise for children with Canavan disease, a rare and fatal leukodystrophy.…
Prostate cancer doesn't affect all men equally. Medical researchers have long known that men with African ancestry experience significantly higher rates and more severe outcomes than other groups. Now, according…
As reported by The Pharma Letter, the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted a new indication for argenx’s Vyvgart (efgartigimod alfa), expanding the therapy’s use to…
Editor's Note: Patient Worthy is proud to share this story from the Courageous Parents Network, originally written by Kelsey Stanczyk. When I first became a nurse, I worked in an…
For decades, doctors have prescribed stimulant medications like Ritalin and Adderall to millions of children with ADHD, operating under the assumption that these drugs directly sharpen attention and concentration. But…
I’ve learned something brutal over the last few weeks: Most people cannot tolerate the reality of a child nearly dying. So they dilute it. They soften it. They reach for…
As reported on Forbes, a class of drugs first introduced to combat HIV is now demonstrating unexpected promise for patients with rare genetic and mitochondrial disorders. Once designed to block…
Takeda Pharmaceutical Company has announced breakthrough results from Phase 3 trials of zasocitinib (TAK-279), an innovative oral treatment for moderate-to-severe plaque psoriasis that offers patients a promising new option for…
As reported on FierceBioTech, Biohaven closed out the holiday period with disappointing news: its investigational Kv7.2/7.3 potassium‑channel modulator, BHV‑7000, did not demonstrate efficacy in a phase 2 clinical trial for…
Editor's Note: Patient Worthy is proud to share this story from our friends at Elephants & Tea. To see the story in its original format, please click here. “Well, good…
Menopause-related insomnia affects approximately one in four women during the menopausal transition, yet it remains significantly underdiagnosed and undertreated. A new analysis published in Maturitas journal and reported by The…
As reported by NewsWise, new clinical study led by investigators at the University of North Carolina at Chapel Hill is offering rare hope for families affected by Hunter syndrome, a…
Axsome Therapeutics has reached a significant regulatory milestone with the US Food and Drug Administration's acceptance of a supplemental New Drug Application (NDA) for AXS-05 as a treatment for agitation…
As reported by Forbes, the U.S. Food and Drug Administration has granted approval to Flow Neuroscience for the first prescription brain stimulation device designed for home use in treating major…
Soleno Therapeutics has achieved a significant milestone in the treatment of a rare genetic disorder, announcing the publication of pivotal clinical study results for VYKAT™ XR in the prestigious Journal…
Editor's Note: Patient Worthy is proud to share this caregiver story submitted to us by Missy Williams. Nine years later, I can still picture the moment I received the news…
ImmunityBio's latest clinical findings reveal a significant development in the ongoing battle against non-muscle-invasive bladder cancer, with three-year follow-up data suggesting that combining Anktiva with standard immunotherapy could fundamentally alter…
Editor's Note: Patient Worthy is proud to share this article, originally written by Katie Tat, from our friends at Elephants & Tea. To see the article in its original format,…
As reported on Science Daily, a recent clinical study suggests that a traditional Chinese herbal remedy may offer a unique advantage in treating major depressive disorder (MDD) while paving the…
FibroBiologics Inc, a clinical-stage biotechnology company, has announced a significant breakthrough in regenerative medicine by submitting a Phase 1/2 Investigational New Drug (IND) application to the US Food and Drug…
Editor's Note: Patient Worthy is honored to share Michelle Patidar's patient story, provided to us by our friends at Elephants & Tea. To see the article in its original format,…
My chronic health journey is super complicated. When I first started experiencing my symptoms I wouldn’t tell anyone what I was feeling. I would wake up feeling very achy, sort…
When pharmaceutical companies develop medications for serious diseases like prostate cancer, few anticipate how those treatments might transform care for entirely different patient populations. Yet, according to FiercePharma.com, Foresee Pharmaceuticals…
Altimmune has released updated 48-week results from its phase 2 trial of pemvidutide, a GLP-1/glucagon dual receptor agonist being developed for metabolic dysfunction-associated steatohepatitis (MASH), demonstrating continued weight loss and…
CRISPR Therapeutics has announced encouraging early results for zugocaptagene geleucel (zugo-cel), its investigational allogeneic CAR T therapy targeting CD19, in both autoimmune diseases and hematologic malignancies. Autoimmune Disease Progress In…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
