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A Historic Breakthrough in Huntington’s Disease: Gene Therapy Offers New Hope
For decades, Huntington’s disease had no treatment that could alter its course. The new AMT 130 gene therapy trial shows promise in slowing disease progression, marking a historic milestone for patients, families, and the broader rare disease community.
The Rare360 Editorial Team
Sep 267 min read
Tragic Updates from Sarepta’s Gene Therapy Programs
Tragic deaths in Sarepta’s gene therapy trials have deeply impacted the rare disease community, raising concerns about liver risks, FDA responses, and treatment access.
The Rare360 Editorial Team
Jul 235 min read
Inside the Orphan Drug Act: Incentives Fueling Rare Disease Therapies
This article breaks down what the ODA is, why it matters, the challenges that remain, and what patients and caregivers need to know to advocate for continued progress in rare disease care.
The Rare360 Editorial Team
Jul 217 min read
Montana’s Medical Experimentation Law: A New Frontier—or a Dangerous Gamble?
Explore Montana's Medical Experimentation law. Is it a groundbreaking step or a risky gamble?
The Rare360 Editorial Team
Jul 113 min read
Custom Gene Editing Saves Infant with Rare Metabolic Disorder
In a groundbreaking first, doctors developed a custom gene- editing therapy in just seven months to treat a baby with a deadly and ultra-rare metabolic disorder.
The Rare360 Editorial Team
Jun 112 min read
FDA's Diversity Guidance Withdrawal Sparks Concerns Over Clinical Trial Inclusivity
In a controversial move, the FDA has quietly removed its draft guidance on clinical trial diversity, sparking concerns over inclusivity.
The Rare360 Editorial Team
Feb 103 min read
The Genetic Puzzle Solved: Why Some People Stay Symptom-Free Despite Disease Genes
Why do some people inherit disease-causing genes but show no symptoms?
The Rare360 Editorial Team
Jan 213 min read
Breakthrough Discovery Uncovers a New Genetic Link to Autism Spectrum Disorder
A groundbreaking study has linked the DDX53 gene, located on the X chromosome, to autism spectrum disorder (ASD).
The Rare360 Editorial Team
Jan 203 min read
Study Unveils Pervasive Ableism in U.S. Mental Healthcare Settings
Ableism and systemic barriers in mental healthcare continue to challenge U.S. adults with disabilities.
The Rare360 Editorial Team
Jan 33 min read
Genetic Data for Sale? The Ethical Dilemma of 23andMe’s Financial Struggles
Once a trailblazer in genetic testing, 23andMe now faces financial uncertainty, sparking concerns about the future of its genetic database.
The Rare360 Editorial Team
Dec 3, 20245 min read
A Major Milestone for Disability Rights: DOT’s Historic Fine on American Airlines
The U.S. DOT recent $50 million fine against American Airlines marks a pivotal moment in the fight for disability rights in air travel.
The Rare360 Editorial Team
Oct 29, 20245 min read
The Overlooked Crisis: How Rare Eye Disease Patients Are Left in the Dark
Millions of adults on Medicaid lack essential coverage for routine eye care, creating a silent crisis for those with rare diseases.
The Rare360 Editorial Team
Oct 25, 20246 min read
The FDA’s Duloxetine Recall: Implications for Mental Health and Chronic Pain Patients
Thousands of Duloxetine Capsules have been recalled after N-nitroso-duloxetine, a cancer-causing impurity, was detected above safety limit.
The Rare360 Editorial Team
Oct 21, 20242 min read
Navigating the Mental Health Coverage Gap in the Rare Disease Community
Rare disease patients face unique mental health challenges, yet many struggle to access care due to insurance coverage barriers.
The Rare360 Editorial Team
Oct 15, 20246 min read
From CRISPR to Jumping Genes: The Next Breakthrough in Genetic Science
New research on "jumping genes" could lead to safer, more precise gene-editing techniques.
The Rare360 Editorial Team
Oct 3, 20244 min read
Medicare Prescription Payment Plan Explained: Lowering Costs for High-Priced Medications
The new Medicare Prescription Payment Plan will help reduce out-of-pocket drug costs and make medications more affordable.
The Rare360 Editorial Team
Sep 24, 20244 min read
FDA Approves First-Ever Epinephrine Nasal Spray (Neffy) for Treating Life-Threatening Allergic Reactions
Neffy, the FDA-approved epinephrine nasal spray, offering a groundbreaking alternative to injections for managing severe allergic reactions.
The Rare360 Editorial Team
Sep 9, 20243 min read
Vision Services Disparity: How Medicaid’s State-Level Policies Affect the US Rare Disease Community
Learn why expanding Medicaid vision coverage is crucial for the rare disease community and what policy reforms could help.
The Rare360 Editorial Team
Sep 9, 20244 min read
Digital Health in Rare Diseases: How Wearables are Transforming PNH Care
Learn how wearable technology, like Fitbits, is helping patients with PNH track their overall well-being.
The Rare360 Editorial Team
Sep 5, 20243 min read
FDA Extends Priority Review Period for Kresladi/ RP-L 201 in Treating Severe Leukocyte Adhesion Deficiency-I (LAD-1)
Explore the FDA's extension of the priority review period for Kresladi, a groundbreaking gene therapy for severe LAD-1
The Rare360 Editorial Team
Feb 19, 20242 min read
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