Rare Disease
Can We Fix This Broken Heart? – Part 1
Editor’s Note: The following is the first part of excerpt written and submitted to us by Dana Langston. There is a specific kind of internal
Alkermes Launches Phase 3 Program for Investigational Narcolepsy Therapy Alixorexton
- April 7, 2026
Can We Fix This Broken Heart? – Part 2
A New Hope for Treatment-Resistant Multiple Myeloma: CellCentric Advances InoPd into Pivotal Clinical Testing
Gilead Ends Midstage HIV Study as FDA Clinical Hold Continues
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Rare Disease
Draft Guidance from FDA Introduces Important Changes to Drug Development for Rare Diseases
On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific
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
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
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HIV unfolds as a dynamic interaction between a virus and the immune system, moving through distinct phases that reflect shifting control over the body's defense. Shortly after exposure, the virus establishes itself rapidly, spreading through immune tissues and embedding into key cells that normally coordinate protection. Over time, even as the body mounts a response, HIV adapts and persists, creating a prolonged period where outward health may appear stable while subtle immune damage accumulates beneath the surface. Understanding this progression highlights why early detection and continuous treatment are essential to preserving immune function and long term health. Click the link below to share your story today - bit.ly/3VuxFDV #hivawareness #hiveducation #KnowYourStatus #PatientWorthy
Sotos syndrome is a rare genetic condition that affects growth and development, often leading to physical overgrowth in childhood along with developmental differences. Because it’s rare, many families may spend time searching for answers before receiving a diagnosis.
Raising awareness helps bring more understanding to conditions like Sotos syndrome and the individuals and families navigating them every day. By learning about rare diseases, we can help create a more informed, supportive community.
#SotosSyndrome #SotosSyndromeAwareness #RareDiseaseOfTheWeek #RareDiseaseAwareness #PatientWorthy
A New Hope for Treatment-Resistant Multiple Myeloma: CellCentric Advances InoPd into Pivotal Clinical Testing bit.ly/4slMlDA Read now at PatientWorthy.com
#PatientWorthy #multiplemyeloma #rrmm #clinicaltrials
