Huntington's Drugs News Monitoring

FDA official calls UniQure's Huntington's disease treatment a failure

By Michael Erman and Puyaan Singh March 5 (Reuters) - A senior FDA official called UniQure's experimental treatment for Huntington's disease a "failed product" in ‌a conference call with media members on Thursday, casting further ‌doubt …

Republican Launches Investigation Into Trump Admin FDA Denials

Senator Ron Johnson has launched an investigation into the Food and Drug Administration (FDA) over the agency's recent denials of treatments for rare diseases, the Wisconsin Republican said in an interview with Bloomberg News. The FDA has rejected …

<![CDATA[uniQure and FDA Clash Over Regulatory Decision for Huntington’s Disease Therapy: Report ]]>

FDA informed uniQure it will not accept data from the company's Phase I/II studies as the primary basis for a marketing application for AMT-130, its investigational gene therapy for Huntington's disease. The decision deals a significant blow to …

Trump administration's embattled FDA vaccine chief is leaving — again

The Food and Drug Administration's embattled vaccine chief, Dr. Vinay Prasad, is once again leaving the agency — the second time in less than a year that he's departed after controversial decisions involving the review of vaccinations and specialty …

Young woman took her own life after more than a decade of fearing she would fall victim to Huntington's disease that ...

A young woman took her own life after more than a decade of living in fear that she would fall victim to Huntington’s disease that killed her grandfather and mother, an inquest heard. Olivia Martinez, 29, had started to undergo tests to see if she would …

New European project develops human-based models for brain disorders

The University of Barcelona is consolidating its position at the forefront of European research thanks to the strategic project "VISI-ON-BRAIN: Cutting-edge Human In Vitro and In Silico Biomedical Tools on Brain Disorders". This is a training and …

FDA Defends Call For Placebo Study In uniQure Huntington's Gene Therapy Trial

An official from the U.S. Food and Drug Administration (FDA) defended the agency's request for a new placebo-controlled study for uniQure N.V.’s (NASDAQ:QURE) experimental gene therapy for Huntington's disease. The FDA official noted that the …

New FDA knockback hits uniQure and Huntington patients hard

uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, as the FDA will not accept a filing based on its current clinical data. Shares in the Netherlands-headquartered company, already depressed by the FDA…

FDA tells uniQure early trial data insufficient for brain disorder therapy filing

Published by Global Banking & Finance Review® Posted on March 2, 2026 1 min read Last updated: March 2, 2026 March 2 (Reuters) - UniQure said on Monday the U.S. health regulator has informed the company that data from an early- to mid-stage trial of …

Orphan Drugs Market 2026-2034: Analysis by Drug & Disease Type, Phase, Top Selling Drugs, Distribution Channel, & Region

The orphan drugs market share is expanding, driven by the increasing knowledge of rare diseases as a major public health issue. BROOKLYN, NY, UNITED STATES, February 25, 2026 /⁨EINPresswire.com⁩/ -- NEW YORK, USA — The global orphan drugs industry is on a …

QURE Or RGNX: Which Gene Therapy Was FDA Chief Really Warning About In Rare Disease Remarks?

Analysts are divided on the target, with RBC Capital and Stifel saying the comments appeared to reference uniQure, while Leerink argued they more closely align with Regenxbio’s RGX-121. FDA Commissioner Marty Makary flagged concerns around invasive gene …

Unlearn Advances Huntington's Disease AI Modeling Through Access to CHDI Foundation Data

SAN FRANCISCO, CA AND NEW YORK, NY / ACCESS Newswire / February 24, 2026 / Unlearn, a leading artificial intelligence company in the clinical development space, today announced its use of data from CHDI Foundation, Inc., a privately-funded …

UniQure says FDA prefers randomized trial for Huntington's treatment

Michigan man gets gene therapy for hemophilia A outside clinical trials

A Michigan man is the first patient in the state, outside of clinical trials, to receive a new gene therapy for hemophilia A at Henry Ford Health, a one-time intravenous infusion that could eliminate the need for regular, lifelong at-home infusions or …

New Alliance Brings Together 1.2 Million Illinois Rare Disease Patients

A chance meeting in a DC hallway turns into a movement SPRINGFIELD, IL, UNITED STATES, March 13, 2026 /⁨EINPresswire.com⁩/ -- In the world of rare diseases, finding someone who understands your journey can feel like finding a needle in a haystack …

"The Cannabis Industry's Medical Pivot: Why Most Companies Are Already Ten Years Behind."

Duane Boise CEO MMJ International Holdings stated "You cannot shortcut the FDA. Many companies are just now deciding they want to be pharmaceutical, but the real barrier to entry is time spent inside the regulatory process. MMJ International Holdings …

Harness Therapeutics steps up fight against Huntington's disease

The Babraham Research Campus company has nominated HRN001 as lead drug candidate for Huntington’s and formed a top-class clinical advisory board to support the programme's advancement towards clinical evaluation. Huntington’s disease (HD) is a …

New CRISPR technique boosts mitochondrial function to treat heart failure

After a heart attack, the heart struggles to recoup and maintain energy. One third of patients develop heart failure as a result ⎯ a condition that impacts 6.8 million Americans and carries a high lifetime risk with 1 in 4 adults in the U.S. expected to …

Actor Will Forte Joins Huntington's Disease Awareness Campaign

Actor-comedian Will Forte has a brother-in-law, Doug, with Huntington’s Disease (HD), and they’re both now starring in an awareness campaign about the rare neurological disease from Teva Pharmaceuticals. HD, probably most known as felling legendary …

Automotive Active Health Monitoring System Market 2026 Smart Vehicle Technologies Enhancing Driver Safety

The Business Research Company's Automotive Active Health Monitoring System Market 2026 Smart Vehicle Technologies Enhancing Driver Safety LONDON, GREATER LONDON, UNITED KINGDOM, March 18, 2026 /⁨EINPresswire.com⁩/ -- "Automotive Active …

LSA Launches SmartCheck: The First Real-Time Insurance Validation Tool for Interpretation Services

LSA SmartCheck is the first real-time insurance validation tool, providing health plans instant coverage checks, enhanced compliance, and better cost control. PA, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- Language Services Associates ( …

Qalitex Laboratories Offers GMP Consulting for 21 CFR Part 111 Quality System Development

GMP Consulting Supports FDA Audits, SOPs, Testing, and GMP Readiness for Supplement Manufacturers IRVINE, CA, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- Qalitex Laboratories, an ISO 17025-accredited testing laboratory with GMP consulting …

Estetica Wellness Medical Spa Opens in Las Vegas

Cutting-Edge Concierge Care, Advanced Aesthetics and Whole-Body Wellness Under One Roof LAS VEGAS, NV, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- Estetica Wellness Medical Spa, a premier Las Vegas destination for medical aesthetics, …

Bath and Shower Products Market: Analysis of Future Demand and Leading Key Players Through 2030

The Business Research Company’s Bath and Shower Products Global Market Report 2026 – Market Size, Trends, And Forecast 2026-2035 LONDON, GREATER LONDON, UNITED KINGDOM, March 18, 2026 /⁨EINPresswire.com⁩/ -- The bath and shower products sector has …

Beauty Devices Market: Size, Share, Competitive Overview, and Trend Analysis Report

The Business Research Company’s Beauty Devices Global Market Report 2026 – Market Size, Trends, And Forecast 2026-2035 LONDON, GREATER LONDON, UNITED KINGDOM, March 18, 2026 /⁨EINPresswire.com⁩/ -- The beauty devices market has been experiencing …

Qalitex Laboratories Achieves ISO 17025 Accreditation for Analytical Testing

A2LA-accredited Irvine lab explains why accredited vs. self-declared compliance determines COA acceptance by Amazon, retailers, and FDA. IRVINE, CA, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- Qalitex Laboratories, an ISO 17025- …

Simply Onno: AI Service That Translates and Explains Medical Documents into Plain Language Now Available in English

Built in Germany with the highest medical quality standards, Onno now brings this service to English speakers. BERLIN, BERLIN, GERMANY, March 17, 2026 /⁨EINPresswire.com⁩/ -- Simply Onno, the AI-powered service to translate medical documents into …

Qalitex Laboratories Expands Pharmaceutical Testing Services for Drug Developers and CROs

ISO 17025-accredited California lab offers HPLC, LC-MS/MS, ICP-MS, and ICH stability studies with 48-hour turnaround for FDA submissions. IRVINE, CA, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- Qalitex Laboratories, an ISO 17025-accredited third …

Qalitex Laboratories Launches Regulatory Consulting for Supplement and Pharma Brands

ISO 17025 Lab Provides FDA, GMP, and Amazon Compliance Support for Supplement & Pharma Brands IRVINE, CA, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- Qalitex Laboratories, an ISO 17025-accredited testing laboratory offering regulatory …

US News & World Reports: UOI’s East Bay Surgery Center Among Nation’s Best ASCs for Orthopedics & Spine

Only Rhode Island ASC to earn repeat recognition; One of 233 orthopedic centers nationwide to be ranked high-performing EAST PROVIDENCE, RI, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- University Orthopedics is proud to announce that for …

Global Walk Assist Robot Market to Reach USD 568.81 Million by 2031 Despite Cost Pressures | Arizton

Industry Analysis Report, Regional Outlook, Growth Potential, Price Trends, Competitive Market Share & Forecast 2026–2031. CHICAGO, IL, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- The global walk assist robot market is gaining …

Interdisciplinary Dental Education Academy (IDEA) Sets the Standard for Advanced Dental Education

Interdisciplinary Dental Education Academy (IDEA) is the original global leader in advanced, hands-on dental education — the only true IDEA in the space. FOSTER CITY, CA, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- The Interdisciplinary Dental …

CST Academy Celebrates Grand Opening of Pediatric Therapy Clinic Near Park Ridge

New clinic near Park Ridge expands access to multidisciplinary pediatric therapy services for families across Chicago’s Northwest Side and surrounding suburbs. PARK RIDGE, IL, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- CST Academy has …

Why Patients Are Traveling to Playa del Carmen for Veneers and Cosmetic Dentistry in Mexico

A1 Smile Design explains the types of dental veneers available in Mexico, their benefits, and why Playa del Carmen is a top destination for cosmetic dentistry. PLAYA DEL CARMEN, QUINTANA ROO, MEXICO, March 17, 2026 /⁨EINPresswire.com⁩/ -- More …

Your Doctors Online Reports Serving More Than 1 Million Patients Through Its Virtual Healthcare Platform

Your Doctors Online says its telehealth platform has now served more than one million patients, reflecting growing demand for virtual medical consultations. TORONTO, ONTARIO, CANADA, March 17, 2026 /⁨EINPresswire.com⁩/ -- Your Doctors Online …

Students Turn Raw News Data Into Visual Stories at 2026 Newsmatics Hackathon in Brno

High school, undergraduate and graduate students competed over 24 hours to analyze news trends, forecast future cycles, and more WASHINGTON , DC, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- Students from across Brno, Czechia spent 24 …

InSkin Laser Aesthetics Introduces the Matrix® Skin Renewal Platform: A Revolutionary Approach to Skin Health

OAKBROOK TERRACE , IL, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- InSkin Laser Aesthetics is proud to announce the addition of the Candela Matrix® platform to its comprehensive suite of aesthetic services. This groundbreaking system is …

Earth Buddy Revisits 2022 Observational Study to Highlight Individual Variance in Pet Wellness

Earth Buddy is taking a look at the findings from its 2022 observational study to openly discuss a reality frequently overlooked in the pet wellness space. LONGMONT, CO, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- Earth Buddy is taking a fresh …

Injury Care Solutions Group: A Well-Known Wide Receiver and the Lisfranc Injury Explained

Dr. Greg Vigna highlights wide receiver's resilience after injury and underscores the value of evidence-based expert analysis in litigation LOS ANGELES, CA, UNITED STATES, March 17, 2026 /⁨EINPresswire.com⁩/ -- Dr. Greg Vigna, Board Certified …