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
Editor’s Note: Patient Worthy is honored to present this story by Heather Doyle, originally published by the United Porphyrias Association. To see the article in
Editor’s Note: This piece, originally written by Kayla DeSonier, was shared with us by our friends at Elephants & Tea. To see the article in
Editor’s Note: This article was shared with us by our friends at the Cure NF2 Foundation, in honor of May 22nd being World NF2 Awareness
Exelixis has taken a major step forward in its colorectal cancer development pipeline by securing supplies of Merck’s Keytruda for an upcoming phase 3 clinical
Betty Vertin is a mother, writer and a fierce defender of the decisions she and her husband Jason made about the seven children they brought
According to the CDC, a look back at April 2026 cumulative reports showed more than 139 pediatric deaths with approximately 250,000 people hospitalized. In simple
Atossa Therapeutics Inc. has achieved a significant regulatory milestone with the U.S. Food and Drug Administration’s grant of Rare Pediatric Disease (RPD) designation to (Z)-endoxifen
Management and treatment of Multifocal Motor Neuropathy (MMN) are less about “fixing” the condition and more about helping people continue living their lives in a way that feels sustainable and meaningful. Over time, many patients learn how to listen to their bodies—pacing energy, adapting routines, and finding new ways to do familiar tasks. With the right support and consistency, these approaches can help maintain strength, reduce day-to-day strain, and protect independence. Even when changes happen gradually, having a plan in place can make those changes feel more manageable and less overwhelming. For more information, visit our website at patientworthy.com
If you have a story to share about your journey with MMN, click the link below!
bit.ly/4dV7gru
#MMN #MultifocalMotorNeuropathy #RareDisease #RareButReal #ShareYourStory
Multifocal Motor Neuropathy (MMN) is a rare neurological condition that primarily affects movement rather than sensation. Because symptoms often begin gradually and may resemble other neuromuscular disorders, understanding the signs can help support earlier recognition and diagnosis. Awareness matters—especially for conditions that many people have never heard of until they or someone they love is affected.
Click the link below to share - bit.ly/3VuxFDV
#mmn #rareneurologicaldisease #PatientWorthy
For many people living with rare diseases, changes in the voice can be more than just hoarseness. Neurological, muscular, and other underlying conditions can affect the ability to speak clearly, communicate comfortably, and be understood by others. Raising awareness of symptoms like these helps highlight the often-overlooked ways rare diseases impact daily life and why every patient deserves to have their voice heard.
#rarewordoftheweek #raredisease #rarediseaseawareness #PatientWorthy
© 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.
