Cancer cachexia represents one of oncology’s most overlooked catastrophes. Patients waste away, lose appetite, and deteriorate despite receiving chemotherapy, yet few treatment options exist to
I had learned that the many twists and turns on our rare disease path were similar to the families we met Originally published on May 11, 2022,
I was born in Long Island, New York, and raised in Southern California, where I grew up surrounded by sunshine and the beauty of the
Diabetic macular edema represents a silent threat to working professionals across America. The condition, which causes fluid accumulation in the macula, the eye’s central focusing
As reported on PharmaBiz, ModeX Therapeutics, a clinical-stage biotechnology company within OPKO Health, has begun dosing patients in a first-in-human clinical trial evaluating MDX2003, a
A New Shield for the Blood Supply: Grifols Earns FDA Approval for Malaria Test Grifols has reached a significant milestone in transfusion medicine with the
The aesthetic skincare market has long overlooked a fundamental principle: skin preparation matters as much as the procedure itself. According to PharmaBiz.com, Galderma’s Alastin brand
As reported on Business Wire, Motif Neurotech has received clearance from the U.S. Food and Drug Administration (FDA) to initiate its first clinical study evaluating
For patients with rare diseases and the caregivers in healthcare who support them, skin changes often become a daily friction point rather than a simple
Pancreatic cancer remains one of medicine’s most formidable challenges, and recent clinical developments suggest a potential turning point. Revolution Medicines presented compelling evidence this week
Early Phase III data suggest C5 complement inhibition may alter disease course in IgAN In a recent press release, AstraZeneca reported that Ultomiris (ravulizumab) has
Yoga is often celebrated for its ability to help people find their “zen,” but new research shows it might be doing some heavy lifting for
AskBio Inc., a gene therapy subsidiary of pharmaceutical giant Bayer AG, has taken a significant step forward in advancing treatment options for Parkinson’s disease. According
For nearly two decades, I’ve been building and maintaining a global online community for individuals and families affected by Moebius syndrome, a rare neurological condition
Fernando Mendoza’s journey to becoming the Las Vegas Raiders’ first overall NFL Draft pick is not a typical underdog narrative, it’s a testament to resilience,
Editor’s Note: Patient Worthy is honored to share this piece from our friends at Elephants & Tea, originally written by Katie Newbaum. To see the
As reported on NBC News, an ophthalmologist with a history of criticizing the U.S. government’s Covid-19 vaccine strategy is emerging as a leading contender to
My name is Maha, and I had the privilege of being my father’s — my Baba’s — caregiver. I’ll be blunt; it was tough. When
#clinicaltrials United Leukodystrophy FoundationTo learn more, visit: www.clinicaltrials.gov/study/NCT05819866
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy (CALYX)
Sponsored by: Minoryx Therapeutics
United Leukodystrophy FoundationJoin us for our upcoming webinar:
Intro to Adaptive Tai Chi: Safe Movement for Every Ability presented by Jen Keller, MS, PT
Sunday, May 31st at 2:00pm EST
Tai chi is a mindful movement practice that improves relaxation skills, mental focus, and physical alignment. With nearly 15 years of tai chi practice and more than two decades as a research physical therapist for people with neurodegenerative disorders, Jen is enthusiastic to offer classes in tai chi that are accessible to all ages, abilities, and physical conditions. Certified by Tai Chi Health (R) to teach programs in Tai Chi Fundamentals Adapted and Tai Chi Prime (evidence-based fall prevention program), Jen will offer an introductory class that can be done in sitting, standing with support (walker, cane, chair for support), or standing without support.
Register here: us02web.zoom.us/meeting/register/taoNU5yMQWaF7f-dZPwiqw?blm_aid=0#/registration
3 Part Series - Watch them all. Link Below
#AlagilleSyndrome #PFIC #bare Rare Disease 360Transition of care is a critical and often complicated part of the rare disease journey.
For patients living with Alagille Syndrome and PFIC, moving from pediatric to adult care can bring major challenges, from navigating a less coordinated healthcare system to learning how to manage complex care more independently.
In partnership with Rare Disease 360 this 3-part CLD Summit video series featuring Dr. Jennifer Vittorio and Dr. Shikha Sundaram takes a closer look at what effective transition of care should involve and why it matters so much.
Part 1: Why transition planning should begin early and how to build readiness over time
Part 2: The unique barriers rare disease patients face when moving into adult care
Part 3: Practical tips, support resources, and how to prepare for the final pediatric visit
These conversations reflect many of the concerns raised at the 2025 Cholestatic Liver Disease Summit, where transition of care was a major topic across panel discussions, research roundtables, and shared patient experiences.
Watch the full 3-part series:
Part 1: zurl.co/6ymBz
Part 2: zurl.co/RKJsw
Part 3: zurl.co/Kxz1b
#AlagilleSyndrome #TransitionOfCare #RareDisease #RareDiseaseAdvocacy #CholestaticLiverDisease #PatientSupport #HealthcareTransition
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