HIV/AIDS
Norway Case Adds Insight Into Long-Term HIV Remission After Stem Cell Transplant
As reported on Healthline, a man in Norway has entered sustained HIV remission following a stem cell transplant, becoming one of a small number of
Fighting for Himself and Others: Wojciech’s Battle Against Crohn’s Disease and More
Pre-Surgery Immunotherapy Rewrites Colorectal Cancer Survival: Zero Recurrence at Three Years
AskBio Advances Parkinson’s Gene Therapy Program With Commercial-Scale Manufacturing
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Leukemia
Ziftomenib: FDA’s Newly Approved Lifeline for Leukemia Patients
Two University of Michigan professors, Jolanta Grembecka and her husband Tomasz Cierpicki, created the newly approved leukemia drug ziftomenib. Its recent FDA approval offers new
Duchenne Muscular Dystrophy
Sarepta Advances Precision Genetic Therapies Toward Full FDA Approval: AMONDYS 45 and VYONDYS 53 Seek Traditional Approval Status
Sarepta Therapeutics has announced a significant regulatory milestone in its efforts to secure permanent FDA approval for two breakthrough Duchenne muscular dystrophy (DMD) treatments. As
Rare Disease
Simple Daily Habits to Boost Your Whole-Body Health and Well-Being
Editor’s Note: Patient Worthy is honored to share this article, originally submitted by Camille Johnson. For chronic disease patients living with rare conditions and long-term
Amyloidosis
Extended Acoramidis Data Show Durable Survival Advantage in ATTR-CM
As reported on Cardiovascular Business, new long-term findings presented at the American College of Cardiology (ACC) 2026 meeting provide compelling evidence that early and continuous
Alzheimer's disease
New Study Reports How Individual Areas of the Brain Age
A pivotal study recently published in the journal Gero Science has revealed new insights into how individual brain regions age and why some are more
Spinal Muscular Atrophy
Optimized Dosing Delivers Enhanced Motor Benefits: FDA Approves High-Dose Spinraza Regimen for Spinal Muscular Atrophy
A decade of accumulated clinical experience with nusinersen has culminated in a significant regulatory advancement. According to Pharma Times Online, Biogen’s newly approved high-dose Spinraza
Wet macular degeneration
Ocular Therapeutix Reports Durable One-Year Outcomes for AXPAXLI in Wet AMD
As reported by Manila Times, Ocular Therapeutix has released additional one‑year results from its Phase 3 SOL‑1 trial evaluating AXPAXLI (formerly OTX‑TKI) for the treatment
Rare Disease
Clinical and Regulatory Updates Highlight Momentum Across Biopharma
As reported on BioPharmaDive, several biopharmaceutical companies reported notable clinical and regulatory developments this week, with investor response reflecting growing confidence in both late‑stage pipelines
Dermatomyositis
Targeted Immunomodulation Transforms Dermatomyositis Management: Phase 3 Brepocitinib Trial Demonstrates Significant Clinical Benefits
Researchers have demonstrated substantial therapeutic benefits for brepocitinib, an oral tyrosine kinase 2/Janus kinase 1 inhibitor, in adults with dermatomyositis, according to findings from a
IgA Nephropathy (IgAN)
Akebia Doses First Volunteers in Phase 1 Study of AKB-9090 for Surgery-Related AKI
Akebia Therapeutics has initiated clinical development of a new investigational therapy aimed at preventing or treating acute kidney injury (AKI) associated with cardiac surgery. The
Triple-negative breast cancer
Triple Negative Breast Cancer: A Breakthrough Treatment
Triple negative breast cancer, the most aggressive form of breast cancer, is under attack. Adelaide University researchers, led by Dr. Theresa Hinkey, report that unlike
Hepatocellular Carcinoma
Combinatorial Immunotherapy Strategy: CatalYm Advances Visugromab in Advanced Liver Cancer
CatalYm has initiated patient enrollment in the GDFATHER-HCC-01 trial, a Phase 2b study investigating visugromab as part of a combinatorial treatment strategy for patients with
Lupus
Litifilimab Shows Promise in Phase 2 Trial for Cutaneous Lupus
Phase 2 data suggest meaningful skin improvement and an acceptable safety profile As reported on Healio, litifilimab, an investigational therapy developed by Biogen, demonstrated encouraging
Bladder cancer
A Smarter Urine Test for Bladder Cancer
As reported on Inside Precision Medicine, Researchers at Stanford have created a new urine test that helps doctors understand how well bladder cancer treatments are
Muscular Dystrophy
Strategic Reassessment: Roche Discontinues Emugrobart in Spinal Muscular Atrophy and FSHD
Chugai Pharmaceutical Co., Ltd. announced that Roche has made the strategic decision to discontinue clinical development of GYM329 (emugrobart), an investigational anti-latent myostatin antibody, for
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Living with epidermolysis bullosa can feel isolating, but your story could be the reason someone else feels understood. At Patient Worthy, we believe every experience matters and has the power to bring comfort, connection, and awareness to others navigating similar challenges. If you’ve been diagnosed with EB, we’d be honored to help share your journey and amplify your voice. Click here to get started: bit.ly/4dV7gru
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We're honored to share the story of Wojciech Biesaga, a patient from Poland who is fighting against severe Crohn's disease, as well as several other diagnoses and complications. He hopes that raising awareness will help others, as well as help himself get the attention and aid that he needs in his country. Read here:
bit.ly/4u7IJGS
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