Congestive Heart Failure
Can We Fix This Broken Heart? – Part 5
Editor’s Note: Patient Worthy is pleased to share the final part in a series of excerpts by Dana Langston. Chapter 36: Holding the Line Knowing
Ultomiris Reduces Proteinuria in Phase III Trial for IgA Nephropathy
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Regular Yoga Practice Improves Cardiometabolic Health
AskBio Scales Up Gene Therapy Manufacturing for Landmark Parkinson’s Disease Trial
Awareness Isn’t the Hard Part — What 18 Years in the Moebius Syndrome Community Has Taught Me
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Primary Biliary Cholangitis
A Breakthrough for the Invisible Affliction: FDA Approves First-Ever Treatment for Cholestatic Itch in Liver Disease
For patients living with primary biliary cholangitis (PBC), a rare autoimmune liver disease, an often-overlooked symptom has plagued their existence, an intense, maddening internal itch
Rare Disease
EMERALD‑3 Trial Shows Benefit of Dual Immunotherapy–Based Strategy in Embolization‑Eligible Liver Cancer
As reported on PharmaBiz, a novel treatment strategy combining dual immunotherapy, targeted therapy, and transarterial chemoembolization (TACE) has demonstrated meaningful clinical benefit for patients with
Congestive Heart Failure
Can We Fix This Broken Heart? – Part 4
Editor’s Note: Patient Worthy is pleased to share part 4 of 5 in a series of excerpts written by Dana Langston. Part 7: Legacy &
Major Depressive Disorder
Breaking Through Depression’s Barriers: Real-World Evidence Demonstrates Spravato’s Long-Term Effectiveness
When standard depression medications fail, patients face a devastating reality. Approximately one-third of people with major depressive disorder do not adequately respond to conventional antidepressants,
Thyroid eye disease
Subcutaneous Tepezza Matches Infusion in Phase 3 Trial, Amgen Reports
As reported on BioPharmaDive, Amgen has announced positive Phase 3 results for a subcutaneous version of its thyroid eye disease (TED) therapy Tepezza, suggesting the
Rare Disease
Can We Fix This Broken Heart? – Part 3
Editor’s Note: Patient Worthy is proud to bring you part 3 of 5 in a series of excerpts written by Dana Langston. Part 3: The
Wet macular degeneration
Next-Generation Eye Treatment OLN324 Demonstrates Superior Performance in Major Clinical Trial
Ollin Biosciences has announced important clinical trial results showing that OLN324, an investigational eye treatment, outperforms an existing therapy in treating two serious retinal diseases
Narcolepsy
Alkermes Launches Phase 3 Program for Investigational Narcolepsy Therapy Alixorexton
As reported on Business Wire, Alkermes plc has initiated a global phase 3 clinical development program to evaluate alixorexton, an investigational oral therapy for people
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
Multiple Myeloma
A New Hope for Treatment-Resistant Multiple Myeloma: CellCentric Advances InoPd into Pivotal Clinical Testing
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
HIV/AIDS
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
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
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#geneticcounseling #GeneticTesting @TessResearchFoundationIt's #NationalDNADay! 🎉
Receiving an SLC13A5 Epilepsy diagnosis involves genetic testing, which looks at a person's DNA to find any changes that may affect health. 🧬
Our Founder & Executive Director, Kim Nye, recently joined genetic counselor, Dr. Lauren Beretich to walk through what’s in a genetic report and how this important information can help guide a family’s next steps. ✍️
📹 Watch the video here: bit.ly/4tcWnrj
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A Cure In Sight Ocular MelanomaNew episode! Outliving the Statistics: A Story About Survivor’s Gratitude | The Eye Believe Podcast
📺 youtu.be/Zc6TF_sRnJ8
🎧 www.podbean.com/eas/pb-agqiv-1aa49f7
What does it mean to outlive the statistics? 💙
In this episode of Living in the Long Middle, we hear from Katie Doble, a metastatic ocular melanoma survivor who is now NED following TIL therapy.
Katie shares an honest and powerful perspective on survivor’s gratitude and survivor’s grief—the complex emotions of doing well while others in the community are not. Her story is a reminder that hope and heartbreak can exist at the same time.
💬 This is a deeply personal topic—if it resonates with you, we invite you to share your thoughts in the comments.
Article from Huffpost: www.huffpost.com/entry/doctor-cancer-clinical-trials-advocacy_n_646515dbe4b0bfd6447f88df
Katie's Blog: futurehappyself.com/
UPMC Blog post: hillman.upmc.com/cancer-care/melanoma-skin/patient-stories/katie-doble
Katie's Instagram: www.instagram.com/ceortman/
Cure Syngap1 #RepurposedDrugs🎤 💻 New #CURESYNGAP1 Webinar with Heather Stone, MPH
🧬 CURE ID: A Treatment Registry of Repurposed Drugs for SYNGAP1
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