Rare Disease
A Small Taste of Freedom in an Unfree World
“Do not go gentle into that good night. Rage, rage against the dying of the light.” –Dylan Thomas One may have thought, after six months
VIS-101 Demonstrates Favorable Safety and Early Efficacy in Wet AMD
- March 23, 2026
Jenny Decker’s Journey: An Update
Imfinzi Achieves European Milestone: First Immunotherapy to Improve Survival in Early Gastric Cancer
New Pain Medicine Seeking FDA Approval
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THE LATEST FROM PATIENT WORTHY
Obesity
Oral GLP-1 Candidate Aleniglipron Shows Robust Weight Loss and Favorable Tolerability in Phase 2 Program
In a recent press release, Structure Therapeutics has reported encouraging topline results from its Phase 2 ACCESS clinical program evaluating aleniglipron, an investigational once-daily oral
Leigh syndrome
New Hope for Leigh Syndrome
As reported on Inside Precision Medicine, scientists in Germany have made an exciting discovery that could help children with a very rare and serious illness
Lung Cancer
Regulatory Fast-Track Transforms Lung Cancer Treatment Timeline; Gene Therapy Withdrawal Signals Market Challenges
The biotechnology sector witnessed a striking demonstration of expedited drug development this week, with Boehringer Ingelheim receiving FDA approval for an innovative lung cancer therapy
Rheumatoid Arthritis
Elevara Advances Oral CDK4/6 Inhibitor into Mid-stage Rheumatoid Arthritis Trial
As reported by Pharma Times, Elevara Medicines has initiated a phase 2b clinical study evaluating ELV001, an investigational oral CDK4/6 inhibitor, in people with rheumatoid
Triple-negative breast cancer
Dark Sweet Cherries Against Breast Cancer
Pictured above: Giuliana Noratto, Ph.D., (left) and Lauren Stranahan, Ph.D., led research showing that compounds in dark sweet cherries may help slow the growth and
Breast Cancer
Japan Advances Targeted Breast Cancer Treatment: Daiichi Sankyo Seeks Approval for Innovative Therapy After Surgery
Daiichi Sankyo has taken a significant step toward expanding treatment options in Japan by submitting regulatory approval for Enhertu, an advanced cancer-fighting drug designed to
Asthma
FDA Clears First Independent Generic of GSK’s Flovent Asthma Inhaler
As reported by FiercePharma, the U.S. Food and Drug Administration has approved the first fully independent generic version of GlaxoSmithKline’s widely used asthma inhaler, Flovent,
Trimethylaminuria
My Journey, My Life, My Story, My Book – Carry’s Experiences with “Fish Odor Syndrome”
You can not see, hear, or touch my rare condition. I STINK! (LITERALLY) I wasn’t sure if I was going to tell my story. But
HIV/AIDS
Scientists Redesign HIV-Fighting Antibodies to Strike Faster: A New Engineering Breakthrough
Researchers have created a fundamentally new class of HIV-fighting molecules by combining two separate antiviral mechanisms into a single engineered protein. Early laboratory results, reported
Psoriatic Arthritis
Bimzelx Outperforms Skyrizi in Psoriatic Arthritis Trial, Though Commercial Impact Remains Unclear
As reported on BioPharmaDive, UCB has reported positive results from a head-to-head clinical trial showing that its anti-inflammatory biologic Bimzelx was more effective than AbbVie’s
Colorectal cancer
Skipping A Colonoscopy Almost Cost Me Everything
Editor’s Note: Patient Worthy is proud and honored to share Jennifer’s story with cystic fibrosis and colon cancer, originally published on the Cystic Fibrosis Research
Seizures
Momentum Builds Across Biotech Pipeline: Seizure Breakthrough, Cell Therapy Revival, and Obesity Medicine Gains
The biotechnology sector experienced significant positive developments this week, with Xenon Pharmaceuticals’ seizure medication achieving a decisive late-stage victory while regulatory pathways accelerated for several
Breast Cancer
GLP-1 Receptor Agonists and Breast Cancer: Promising Signals, Pressing Questions
As reported on Healio, glucagon-like peptide-1 (GLP-1) receptor agonists, best known for treating diabetes and obesity, are emerging as unexpected players in breast cancer care.
Triple-negative breast cancer
Study: Engineered Immune Cells and Targeted Treatments May Slow the Spread of Triple-Negative Breast Cancer
CAR T- cells are immune cells that are engineered in a laboratory to attack cancer. The therapy has worked as planned for certain blood cancers
BRCA-mutated Breast Cancer
Breakthrough DNA Repair Inhibitor Advances Rapidly Toward Clinic: Artios’ ART6043 Earns FDA Fast Track Status for Resistant Breast Cancer
Artios Pharma has achieved a significant milestone in cancer treatment development with the U.S. Food and Drug Administration’s granting of Fast Track designation to ART6043,
Multiple Myeloma
Bristol Myers Squibb Reports Positive Phase 3 Data for Mezigdomide-Based Oral Regimen in Relapsed or Refractory Multiple Myeloma
As reported on drugs.com, Bristol Myers Squibb (BMS) has announced encouraging interim results from the Phase 3 portion of the SUCCESSOR-2 clinical trial evaluating oral
Hunter Syndrome
FDA Explains Rejection of Regenxbio’s Hunter Syndrome Gene Therapy
As reported on FierceBioTech, the U.S. Food and Drug Administration has released its complete response letter (CRL) detailing why it declined to approve Regenxbio’s experimental
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Cure GRIN FoundationThe countdown to Virtual GRICON26 is ON!
We’ve built this year’s agenda to bridge the gap between lab research and your daily GRI life. Best of all? Registration is pay what you can! What’s in store:
🔬 Clinical Updates: Phase 3 breakthroughs from industry leaders.
🏠 Living GRI: IEP advocacy, behavior management, and adulthood transitions.
🚀 The Next Frontier: Deep dives into ASOs and genetic therapies.
🤝 Community: Dedicated networking for families and siblings.
Your Saturday, Your Way: Customize your day with breakout sessions that matter most to your journey. HUGE thanks to our Diamond Sponsor, GRIN Therapeutics!
🔗 Register & View Agenda: bit.ly/49nBinf
❤️ Support the Journey
Want to honor a loved one? Become a Family Sponsor! While GRICON is free to attend, your sponsorship ensures a world class experience and fuels the research collaborations our community needs.
✨ Become a Family Sponsor: bit.ly/4sPm3Ks
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