According to a recent article on PharmaBiz, HAYA Therapeutics has announced the completion of dosing in the first cohort of its phase 1 clinical study
According to a recent article in The Manila Times, Jade Biosciences has begun dosing participants in a Phase 2 clinical study, known as JUNIPER, evaluating
An article from Medscape highlighted recent advances in treatment that are reshaping the clinical approach to epidermolysis bullosa (EB), transitioning care from largely supportive management
A recent article by BioPharmaDive highlighted a mid-stage clinical trial that evaluted an investigational Parkinson’s disease therapy co-developed by Biogen and Denali Therapeutics, and that
Johnson & Johnson announced FDA approval of a supplemental new drug application for Caplyta (lumateperone) for the prevention of relapse in schizophrenia, based on compelling
A growing body of research highlights the broader benefits of human papillomavirus (HPV) vaccination beyond its long-standing focus on cervical cancer prevention. A recent large-scale
According to a recent article by The Manila Times, Design Therapeutics has reported encouraging interim results from its ongoing Phase 1/2 RESTORE-FA study, highlighting both
Novartis and PTC Therapeutics have advanced their Huntington’s disease candidate, votoplam, into Phase 3 development following promising mid-stage results. The decision reflects confidence in the
As reported on MedicalXpress, a newly developed RNA-focused analytical method may improve the detection and characterization of repeat expansion disorders, a group of genetic conditions
A recent article from The Manila Times highlighted findings from the ongoing IMPACT‑TD Registry underscore a persistent gap in diagnosing tardive dyskinesia (TD), particularly among
BridgeBio’s Attruby (acoramidis) is positioning itself as a formidable competitor in the transthyretin amyloid cardiomyopathy (ATTR-CM) market, with new pivotal trial data and indirect comparisons
A recent article from SciTechDaily describes the promising approach instituted at a private, non-profit biomedical research firm in San Francisco that incorporates data science, AI,
According to a recent press release, Artios Pharma has begun dosing patients in a global Phase 2 clinical trial evaluating its investigational DNA polymerase theta
A significant advancement in antiviral therapy has emerged with the successful development of a CRISPR-Cas13d-based system designed to combat hepatitis E virus (HEV) infections. This
In a recent press release from Pfizer, it was shared that the European Commission (EC) has broadened the approved use of Pfizer’s marstacimab (brand name
A recent report by the Manila Times highlighted Madrigal Pharmaceuticals’ unveiling of a series of new analyses and real-world findings supporting the therapeutic profile of
UniQure has announced plans to submit its groundbreaking Huntington’s disease gene therapy, AMT-130, for regulatory approval in the United Kingdom later this year, marking a
According to a recent report on MedTechDive, Guardant Health has received U.S. Food and Drug Administration (FDA) approval for an updated version of its liquid
Some rare diseases are visible. Others can spend years hiding behind symptoms that seem unrelated. Wilson disease is one example of why listening to your body, asking questions, and continuing to seek answers can be so important. Every diagnosis starts with a story that deserves to be heard.
#wilsondisease #Wilsondiseaseawareness #rarediseaseoftheweek #raredisease #PatientWorthy
From our friends Periodic Paralysis AssociationWe’re excited to share our first Speaker Spotlight and Topic Tuesday for Periodic Paralysis Conference 2026! Meet Dr. Tyler Nelson, PhD, neuroscientist at the University of Florida, pain researcher, and Periodic Paralysis Association Board Member.
Combining lived experience with cutting-edge research, Dr. Nelson is helping change the conversation around pain in periodic paralysis. Living with hyperkalemic periodic paralysis himself, he brings a unique perspective to his work, ensuring that the patient perspective helps shape research.
At Periodic Paralysis Conference 2026, Dr. Nelson will present: Uncovering Pain Mechanisms in Skeletal Muscle Channelopathies
Dr. Nelson’s research focuses on understanding how muscle hyperexcitability in disorders such as periodic paralysis and myotonic disorders can lead to changes in the nervous system that drive persistent pain. Using electrophysiology, genetics, and behavioral research, he is tackling a question many in our community know all too well: Why does this disease hurt, and what can we do about it?
After earning his PhD in Neurobiology from the University of Pittsburgh School of Medicine and training in the Pittsburgh Center for Pain Research, Dr. Nelson shifted his focus toward periodic paralysis after seeing firsthand how the condition impacts both himself and several members of his family.
Passionate about ensuring overlooked symptoms like pain are taken seriously, his goal is to advance research that leads to better recognition, better treatments, and a higher quality of life for people living with periodic paralysis.
This is a session you will not want to miss! Join us in-person to meet Dr. Nelson and help further his research on pain and periodic paralysis!
📅 October 10–11, 2026
📍 Hilton Orlando Lake Buena Vista – Disney Springs Area
🔎Ask Dr. Nelson about Pickles! 🕶️
Register today at PeriodicParalysis.org/conference-2026
#PeriodicParalysis #chronicpain #InvisibleIllness #ChronicIllness #Conference
From our friends at the LGS Foundation JOIN NOW💡 Did you know that joining the LGS-CORE Study is completely free?
There is no cost to patients or families to participate, and you won't be asked for payment at any point.
Your only investment is your story, and that story has the power to change the future of LGS care. 💜
⏱️ Remember: Signing up only takes about 15 minutes, and you can complete all surveys at your own pace. This means you can return to your session any day without losing your progress.
👉 Learn more and register: www.lgsfoundation.org/lgs-registry/
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#LGSFoundation #LennoxGastautSyndrome #LGS #RareDisease #PatientRegistry
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