Rare Disease
Can We Fix This Broken Heart? – Part 2
Editor’s Note: This is the second part in a series of excerpts written by Dana Langston. Chapter 4: The Internal Shift We are living in
Roche’s Elecsys NfL Blood Test Gains CE Mark for Monitoring Neuroinflammation in MS
- April 23, 2026
Invading the Blood Brain Barrier: FDA Approves Pembrolizumab Combo
Lynk Pharmaceuticals Achieves Regulatory Milestone: Zemprocitinib Advances in China for Atopic Dermatitis Treatment
5 Lessons Nature Taught Me About Living with Pain and Dystonia
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Multiple Myeloma
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Multiple myeloma, a cancer of plasma cells in the bone marrow, has become an increasingly manageable disease over the past two decades, thanks to successive
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Gilead Ends Midstage HIV Study as FDA Clinical Hold Continues
As reported on FierceBioTech, Gilead Sciences has closed one of its midstage clinical studies evaluating an experimental HIV regimen, following ongoing safety concerns that prompted
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
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Draft Guidance from FDA Introduces Important Changes to Drug Development for Rare Diseases
On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific
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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
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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
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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
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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
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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
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Duchenne muscular dystrophy (DMD) is more than a diagnosis—it’s a genetic condition that gradually weakens the muscles over time. It begins with a mutation that prevents the body from producing dystrophin, a protein muscles need to stay strong and protected. Without it, everyday movements slowly become more difficult.
Early signs often show up in childhood and may include frequent falls, trouble running or climbing stairs, difficulty getting up from the floor, and enlarged calf muscles. As the condition progresses, muscle weakness spreads and can eventually affect the heart and lungs.
Raising awareness means recognizing the signs, understanding the cause, and supporting those impacted. Knowledge helps drive earlier diagnosis, better care, and stronger advocacy for treatments and research. #DMDAwareness #MuscularDystrophy #PatientWorthy
Today is Global CRSwNP Awareness Day 2026. Chronic Rhinosinusitis with Nasal Polyps (CRSwNP) is a chronic inflammatory condition where soft growths develop in the nasal passages and sinuses, often causing congestion, reduced sense of smell, facial pressure, and difficulty breathing. Today, we raise awareness and support better understanding and care for those affected! #PatientWorthy #CRSwNP #crswnpawarenessday #NasalPolyps
April 23 is Fibrodysplasia Ossificans Progressiva (FOP) Awareness Day.
FOP is an ultra‑rare genetic condition where bone forms in places it shouldn't, gradually limiting movement. Awareness helps prevent misdiagnosis, supports research, and reminds those living with FOP that they are not alone. 💙
#FOPAwarenessDay #cureFOP #rarediseaseawareness #UltraRare #PatientWorthy
