As reported on PharmaBiz, AstraZeneca’s phase III LATIFY trial has fallen short of its primary objective, with the combination of the ATR inhibitor ceralasertib and
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,
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
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),
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
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
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
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
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
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
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,
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
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
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 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
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™
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
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
Every rare diagnosis has a beginning. One person's journey can spark awareness, lead families toward answers, and help light the path for others who are still searching. Awareness starts somewhere, and often, it starts with one. #rarewordoftheweek #RareDisease #rarediseaseawareness #PatientWorthy
An amazing thing- patients owning their own data, and deciding how to share it for research for the benefit of all. Congratulations! National MPS SocietyWe’re excited to announce a patient-owned and controlled, independent registry for mucopolysaccharidoses (MPS)! Sanofi is proud to provide the initial funding for this Registry, and to support linkage to patient data from its historic MPS I registry into the new National MPS Society Registry when appropriate patient consents are in place. 💜
“This is more than a transfer of data—it is a recognition that behind every registry entry is a child, an adult, or a family navigating the complexities of rare disease. Their voices matter. Their experiences matter. And now, they will have ownership and control over how their stories contribute to scientific progress.” — Terri Klein, President & CEO, National MPS Society
🔗 Read the full announcement: mpssociety.org/en/news/mps-registry-2026/
#MPS #HurlerSyndrome #HunterSyndrome #Sanfilippo #MorquioSyndrome #MaroteauxLamySyndrome #SlySyndrome #HyaluronidaseSyndrome
Obesity has many causes—genetic predisposition, hormonal imbalances, medication side effects, environmental factors, stress, and more. It's not a simple issue with a simple answer. Understanding the complexity is the first step toward real solutions. #obesity #symptoms #PatientWorthy
#shareyourstory here: bit.ly/4dV7gru
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