As reported on PharmaBiz, AtaiBeckley has announced a key milestone in the development of its investigational depression therapy, VLS-01, with the final participant now dosed
Editor’s Note: Patient Worthy is honored to share this story, originally written by Anne and Jerry van Wyk, and shared with us by our friends
Every day, parents just like me sit in doctors’ offices searching for answers. They know something isn’t quite right. Maybe their child isn’t meeting milestones.
As reported on Vermont Biz, researchers at the University of Vermont (UVM) and biotechnology company RS Oncology have reported encouraging results from an early-stage clinical
For more than a century Alzheimer’s disease has been thought to be incurable. But a recent study published in the New England Journal of Medicine
Editor’s Note: This is part 1 of a 3-part caregiver story, submitted to us by Joan Foster, who was a caregiver for her husband Charles’
As reported by The Manila Times, the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to Elevar Therapeutics regarding its
As covered by Fairfax Times, Novo Nordisk has reported encouraging long-term findings for its investigational hemophilia A therapy denecimig (Mim8), with interim results from the
Our friends at Heal Canada have launched the newest edition of their magazine, E3 Advocacy! Their digital publication focuses on the importance of patient advocacy,
As reported on MobiHealth News, the U.S. Food and Drug Administration (FDA) has awarded Breakthrough Device designation to Aurenar’s V-Link System, a noninvasive neuromodulation technology
Can you take me back to when you first became aware that you had a health condition as a child. Did you overhear the adults
As reported on PharmaBiz, Merck has announced a new agreement with the ADAP Crisis Task Force (ACTF) aimed at improving access to its recently approved
As reported on MedPage Today, large retrospective study suggests that glucagon-like peptide-1 (GLP-1) receptor agonists may offer broad health benefits for patients with hidradenitis suppurativa
As reported on BioSpace, Sarepta Therapeutics has reached an important regulatory milestone in its effort to secure traditional FDA approval for two Duchenne muscular dystrophy
As reported on Tech Times, Sanofi has reported positive results from its Phase 3 Baby-COMET study evaluating Nexviazyme (avalglucosidase alfa) in infants with infantile-onset Pompe
Editor’s Note: Patient Worthy is honored to share this interview, featuring Kate, an ovarian cancer survivor, and Dr. Premal Thaker, M.D. “There is fear, and
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has approved Orca Bio’s Tregzi, a precision-engineered cell therapy designed for use in matched-donor
As reported on FiercePharma, the U.S. Food and Drug Administration (FDA) has approved an expanded indication for Vertex Pharmaceuticals’ gene-editing therapy Casgevy (exagamglogene autotemcel), allowing
Sarcoma Awareness Month is observed each year in July to raise awareness of sarcoma, a rare group of cancers that develop in the bones or soft tissues of the body, including muscle, fat, blood vessels, nerves, tendons, and connective tissue.
The goals of Sarcoma Awareness Month include:
• Increasing public awareness and education about sarcoma.
• Encouraging earlier recognition and diagnosis.
• Supporting patients, survivors, caregivers, and families.
• Advocating for increased research funding and improved treatment options.
• Amplifying patient stories and experiences.
Because sarcoma is considered rare, it is often misdiagnosed or diagnosed later than more common cancers. Awareness efforts help educate healthcare providers and the public about symptoms, the importance of specialized care, and the need for continued research.
#SarcomaAwarenessMonth #sarcomaawareness #sarcoma #rarecancer #cancerawareness #patientadvocacy #PatientVoice #caregiversupport #shareyourstory #PatientWorthy 💛
The people who make the biggest difference aren't always the ones with all the answers.
Living with a chronic illness or rare disease often changes the way you see the people around you. It highlights the ones who listen, check in, remember the little things, and continue showing up through every high and low.
Sometimes, the smallest acts of support leave the biggest impact!
#chronicillness #raredisease #chronicillnessawareness #rarediseaseawareness #invisibleillness #youarenotalone #PatientWorthy
GRIN Therapeutics is continuing to expand enrollment for its Phase 3 Beeline study of Radiprodil in children and young adults with Gain-of-Function (GoF) variants in GRIN1, GRIN2A, GRIN2B, or GRIN2D. The multinational study is recruiting participants ages 1 month through 18 years at sites in multiple countries, including numerous locations across the United States.
A recent update to the ClinicalTrials.gov listing now includes contact information for many participating study sites, making it easier for interested families to reach out directly. While not every site has posted contact details yet, additional information is expected to be added over time.
The study includes two groups:
• Participants with qualifying seizures, and
• Participants with significant gain-of-function GRIN-related symptoms who have few or no qualifying seizures.
To learn more about eligibility, view participating sites, and access contact information, visit the ClinicalTrials.gov listing here.
Cure GRIN Foundation
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