Niemann-Pick Type C Disease
An Excerpt from “A Ribbon for Your Hair”
Editor’s Note: The following in from author Stephen Policoff, whose book A Ribbon for Your Hair is out today! Introduction When Stephen Policoff’s adopted daughter
Dizal’s Zegfrovy Delivers Phase 3 Breakthrough in EGFR Exon 20–Mutant NSCLC
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The Luck of the Irish: A Parable of Inheritance, Illness, and Hope
FDA Approves RSV Vaccine for Younger Adults: What You Need to Know
FDA Expands GSK’s Arexvy Approval to Younger Adults at Elevated RSV Risk
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Glioma
Telix Pharmaceuticals Advances Brain Cancer Imaging with European Regulatory Submission
A New Solution to Address Critical Gaps in Glioma Diagnosis and Management Telix Pharmaceuticals, a biopharmaceutical company specializing in therapeutic and diagnostic radiopharmaceuticals, has taken
Wet macular degeneration
VIS-101 Demonstrates Favorable Safety and Early Efficacy in Wet AMD
As reported on Healio, a novel biologic targeting two key angiogenic pathways has shown encouraging early results for the treatment of wet age-related macular degeneration
Charcot-Marie-Tooth disease
Jenny Decker’s Journey: An Update
Editor’s Note: This story is a continuation of Jenny Decker’s global sailing voyage. To see the original article, please click here. Many of our Patient
gastric cancer
Imfinzi Achieves European Milestone: First Immunotherapy to Improve Survival in Early Gastric Cancer
AstraZeneca has received European Commission approval for Imfinzi (durvalumab) in combination with FLOT chemotherapy, a landmark achievement representing the first immunotherapy-based regimen to demonstrate survival
Rare Disease
New Pain Medicine Seeking FDA Approval
People with rare and chronic disease often suffer from long term pain. New ways to alleviate this pain are needed. According to a press release
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
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
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