Cervical Dystonia
How I Manage My Pain – Pain Series Part 3 of 3
This is Part 3 of three articles I wrote about pain. The first 2 are entitled: Part 1- The real impact of pain that people
A New Hope for Treatment-Resistant Multiple Myeloma: CellCentric Advances InoPd into Pivotal Clinical Testing
- April 7, 2026
Gilead Ends Midstage HIV Study as FDA Clinical Hold Continues
Can We Fix This Broken Heart? – Part 1
Draft Guidance from FDA Introduces Important Changes to Drug Development for Rare Diseases
LISTEN TO OUR PODCAST
THE LATEST FROM PATIENT WORTHY
Amyloidosis
Ron’s Fight Against Amyloidosis
My journey with cardiac amyloidosis started shortly after I retired as a firefighter/paramedic in spring 2018. I started with trigger fingers—one in 2018 and two
Cancer
A Promising Approach to Tumor Ablation: Histotripsy
While surgery has long been the standard for removing solid tumors, a revolutionary technique called histotripsy is offering an alternative, as reported on MedPage Today.
Adenoid Cystic Carcinoma
Remix Therapeutics Advances Breakthrough Cancer Therapy with FDA Fast Track Designation for REM-422
Remix Therapeutics has achieved a significant regulatory milestone with the U.S. Food and Drug Administration granting Fast Track designation to REM-422, a first-in-class small molecule
Erythropoietic Protoporphyria
The Sun Sucks, But You Do Not
My whole life growing up, there was this specific buzzing sound that would come alive on the sunniest of days. I’ve been told over the
Pulmonary Embolism
Rachel’s DVT and Pulmonary Embolism Experience
On 12/12/2025, I was admitted into the ER for a thrombectomy due to a bilateral pulmonary embolism. While I am overweight and sometimes eat like
Narcolepsy
Bridging Nutrition Science and Patient Care: ANA and Jazz Pharmaceuticals Launch Cardiovascular Health Initiative
The American Nutrition Association (ANA) has announced a collaborative initiative with Jazz Pharmaceuticals designed to advance evidence-based nutritional education for individuals living with narcolepsy and
Epidermolysis Bullosa
The Mentor She Wished She Had – How Elizabeth Became a Lifeline for EB Families
This patient story is sponsored by Chiesi Global Rare Diseases and is promoted through the Patient Worthy Collaborative Content program. We only publish content that
Rare Disease
How to Build a Lasting Legacy While Living with Chronic Illness
Patients living with chronic or rare diseases and the caregivers coordinating appointments, medications, and daily life often carry a quiet question alongside the medical work:
Dravet Syndrome
The Dravet Syndrome Foundation of Spain’s Annual Meeting 2026
The Dravet Syndrome Foundation of Spain hosted its 2026 Annual Family Meeting on March 20 at the Rafaela Ybarra School in Madrid, bringing together roughly 100
Hunter Syndrome
Breaking Through the Rare Disease Barrier: FDA’s Historic Approval of Denali’s Hunter Syndrome Treatment
The pharmaceutical industry celebrates a significant victory for rare disease patients as the Food and Drug Administration approved Denali Therapeutics’ groundbreaking enzyme replacement therapy for
Obesity
FDA clears higher‑dose Wegovy to support long‑term weight management
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has approved Novo Nordisk’s higher‑dose formulation of semaglutide, Wegovy HD (7.2 mg once weekly),
Multiple Myeloma
Finding My Way Forward: Living with Multiple Myeloma
I used to be really active: swimming, Tai Chi every week, practicing yoga. So, when I first felt persistent rib and back pain in late
Primary Biliary Cholangitis
Lynavoy Achieves FDA Approval: A Breakthrough Treatment for Cholestatic Pruritus in Primary Biliary Cholangitis
GSK has announced a significant advancement in liver disease treatment with FDA approval of Lynavoy (linerixibat), marking the first medication specifically approved in the United
Heart disease
Choosing to Be Me: Redefining My Life with Rheumatic Heart Disease
My diagnosis of rheumatic heart disease came on suddenly. I remember being 12, sitting at the doctor’s office, and was told that I have a
Huntington's disease
Major Breakthrough: Discovery of a Pathway that Allows Brain Cells to Move Toxic Material in Huntington’s Disease
Researchers at Florida Atlantic University (FAU) have identified a cellular pathway that allows brain cells to share proteins and other toxic material. The study was
Narcolepsy
Lupin Receives FDA Approval for Pitolisant: A Novel Solution for Narcolepsy Management
Lupin, a globally recognized pharmaceutical company, has achieved a significant regulatory milestone with the receipt of tentative FDA approval for Pitolisant tablets in strengths of
Diabetes Type 2
Can Your Morning Coffee Really Help Prevent Type 2 Diabetes?
Editor’s Note: Patient Worthy is honored to present this article, shared with us by our friends at the Steatotic Liver Foundation (formerly the Fatty Liver
FEATURED
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
Bohring-Optiz Syndrome (BOS) is a rare genetic disorder that impacts growth and development, often associated with feeding difficulties, developmental delays, and distinct physical characteristics. While uncommon, it has a profound impact on individuals and families within the BOS community.
Observed annually on April 6th, Bohring-Optiz Syndrome Awareness Day is dedicated to increasing understanding, supporting affected families, and advancing research. By raising awareness, we help foster greater recognition, compassion, and process toward improved care and outcomes.
Share your rare disease journey and inspire others here: bit.ly/4dV7gru
#bohring-OptizSyndrome #BOSAwarenessDay #PatientWorthy
Throughout the week, Patient Worthy is highlighting HIV with educational content aimed at raising awareness and fostering greater understanding in our community.
We’ll be sharing information on our socials and patientworthy.com to educate, empower, and support those affected.
If you'd like to share your story with HIV, click here: bit.ly/4dV7gru
#HumanImmunodeficiencyVirus #HIV #HIVAwareness #ShareYourStory #PatientWorthy
April 5th is Barth Syndrome Awareness Day - a time to shine a light on a rare genetic condition that affects the heart, muscles, and immune system. Barth Syndrome may be a little known, but for those living with it, the challenges are very real. Raising Awareness helps drive research, early diagnosis, and support for families who need it most.
Every voice matters. By learning, sharing, and advocating, we can help create a future where rare diseases like this are better understood and treated. #barthsyndromeawareness #geneticdisorders #PatientWorthy
