As reported on MedPage Today, an oral targeted therapy may offer a safe and effective alternative to injectable treatment for children with achondroplasia, according to new phase II data published…
Editor's Note: Patient Worthy is honored to share this post from Martha Harlam, Director of the Solution Project. Are you a student or researcher passionate about the future of neurology?…
In the whirlwind of early motherhood, days blur together in a haze of feedings, diaper changes, and sleepless nights. You’re learning your baby’s every sound, every expression, and trying to…
A radically new type of optical sensor can identify individual cancer-related proteins in blood, a capability that could revolutionize how early-stage breast cancer is diagnosed and treated. according to Nanowerk.com,…
As reported on Business Wire, ImmunityBio has released new clinical findings suggesting that its interleukin‑15 superagonist, ANKTIVA® (nogapendekin alfa inbakicept), may help restore immune function and improve survival outcomes in…
The U.S. Food and Drug Administration (FDA) has approved Yartemlea (narsoplimab‑wuug), marking the first authorized therapy for hematopoietic stem cell transplant‑associated thrombotic microangiopathy (TA‑TMA) in adults and children aged 2…
Young cancer patients with advanced Hodgkin lymphoma now have access to a significantly more effective treatment option that dramatically reduces their need for radiation therapy and offers superior long-term survival…
As reported by Technology, a major evidence synthesis published in The BMJ has delivered the clearest picture to date of which therapies offer the strongest short‑term benefits for people with…
Editor's Note: Patient Worthy is honored to bring you this article, originally written by Kelsey Stanczyk, and shared with us by our friends at the Courageous Parents Network. So much…
IgA nephropathy represents one of the most pressing kidney health challenges facing young adults worldwide. This progressive kidney disease develops when the immune system malfunctions, leading to kidney damage that…
This podcast episode is sponsored by AVEO Oncology. The product information in this podcast is intended only for US residents. This episode features a real patient and their care partner,…
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 the PD‑L1 inhibitor Imfinzi (durvalumab)…
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…
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