Parabilis Medicines has achieved a significant milestone in cancer drug development, receiving FDA Fast Track designation for FOG-001, a groundbreaking first-in-class therapy targeting desmoid tumors.
Breaking a system into small independent and interchangeable modules that can perform certain functions is called modularity. For many years the KRAS oncogene proved to
As reported on MedicalXpress, esearchers at the Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania are pioneering a new approach to treating rare
In a significant development for neurodegenerative disease treatment, BioPharma Dive reports that AC Immune has unveiled compelling evidence that its novel immunological strategy may fundamentally
An experimental cancer vaccine from the Johns Hopkins Kimmel Cancer Center and the Bloomberg~Kimmel Institute for Cancer Immunotherapy has delivered encouraging results in a phase
Chiesi Group has entered an exclusive licensing agreement with Aliada Therapeutics, a subsidiary of AbbVie, to develop therapies that can cross the blood-brain barrier (BBB)
As reported on Healio, new findings presented at The Liver Meeting highlight the potential of resmetirom (Rezdiffra, Madrigal Pharmaceuticals) in managing metabolic dysfunction-associated steatohepatitis (MASH)
Researchers at the University of Southampton have uncovered a hidden culprit in blood cancer, a unique sugar molecule that disguises aggressive lymphoma cells and renders
A new artificial intelligence model, popEVE, could accelerate diagnoses for rare, single-variant genetic diseases by scoring how likely each genetic change in a patient’s genome
University of Southampton researchers have identified a distinct subtype of diffuse large B‑cell lymphoma (DLBCL), termed “Mann-type DLBCL,” marked by a unique mannose sugar on
Light chain amyloidosis (AL) is a progressive and rare disease whereby abnormal plasma cells found in the blood and urine over-produce light chain proteins. These
The findings were recently published in Nature Immunology and reported by Precision Medicine suggesting that a factor called Interferon Regulatory Factor 4 (IRF4) takes responsibility
As reported on NPR, Tatiana Schlossberg, journalist and granddaughter of former U.S. President John F. Kennedy, has disclosed that she is battling a rare and
As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has launched an investigation into reports of serious adverse events, including one fatality, associated
Three-year-old Oliver Chu has stunned clinicians after becoming the first person with Hunter syndrome (MPS II) to receive an experimental gene therapy and showing striking
Editor’s Note: Patient Worthy is pleased to share this article from our friends at Elephants & Tea, originally written by Cecily Liu. To see the
The United Leukodystrophy Foundation (ULF) is proud and excited to announce a partnership with Marc Nolan, a Chicago-based footwear brand, who is launching a new
The U.S. Food and Drug Administration (FDA) has approved Koselugo (selumetinib), an oral MEK inhibitor developed by Alexion, AstraZeneca Rare Disease, for adults with neurofibromatosis
#mytonicdystrophy #clinicaltrialResearchers at Sanofi are recruiting adults with non-congenital myotonic dystrophy type 1 for a phase 1/2 clinical trial evaluating an investigational gene therapy. 🧬💪
The study will assess the safety of SAR446268 and its potential to target the toxic RNA that drives DM1. Participants will receive a single IV infusion and be followed over two years.
Learn who may be eligible and what participation involves. 👉 mdaquest.org/clinical-research-alert-phase-1-study-of-gene-therapy-in-individuals-with-myotonic-d...
#MyotonicDystrophy #DM1 #ClinicalTrials #GeneTherapy #RareDisease #Research
@csnk2a1foundation.org What a line-up of support ! Mark your calendars today.!🗓️ SAVE THE DATES! 🗓️
Join us throughout 2026 for our bi-monthly OCNDS Family Zoom Meetings hosted by our Parent Advisory Board. Together, we’ll discuss successes, challenges, and all things OCNDS.
👉 Please note: These meetings are only for primary caregivers, guardians, and those living with OCNDS.
👉 We will be using AI technology for live-caption translation—most languages are supported.
👉 Meetings are always held at 08:00 AM Pacific Time (US & Canada) over Zoom.
2026 Family Zoom Meeting Dates
Sunday, February 8 – loom.ly/YOMNi9Q
Saturday, April 18 – loom.ly/-vFIho4
Sunday, June 14 – loom.ly/vONpS7k
Saturday, August 15 – loom.ly/gi5gNj4
Sunday, October 25 – loom.ly/V5ACR8g
Saturday, December 12 – loom.ly/_0jobm8
After registering, you will receive a confirmation email with details for joining the meeting.
💙💚 We look forward to connecting with you throughout 2026!
#ocnds #csnk2a1 #hopeintoaction #timeisnow #BoldBreakthroughs
@CureDucheneOne of the most anticipated experiences of FUTURES 2026 is back: the Discovery Cove dolphin swim.💙
Secure your spot for our exclusive Discovery Cove Family Excursion! Space is limited, and early registration gives you the best chance to enjoy this unforgettable Orlando experience. (Dolphin interactions are limited and not guaranteed — but registering now gets you in line!)🤩
Register now: web.cvent.com/event/ee9e7402-2c59-420f-9cf9-5731cf20d4d9/summary
#dmd#duchenne#dolphin#orlando
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