Duchenne News Monitoring

Solid Biosciences Stock Pops On US, Canada Nod For Gene Therapy Trials: Retail Buzz Spikes

The company plans to begin a Phase 1b trial of SGT-501 later this year, targeting CPVT, a rare and potentially fatal arrhythmia in children and young adults with no current FDA-approved therapies. Solid Biosciences drew heightened retail chatter on Tuesday …

Otsuka’s Taiho reports Phase 3 fail for Duchenne muscular dystrophy drug

Taiho’s DMD therapy misses the mark in Phase III trial

Taiho Pharmaceutical’s investigational Duchenne muscular dystrophy (DMD) therapy, pizuglanstat, has shown no benefit in a Phase III trial. The REACH-DMD study (NCT04587908) showed no significant difference in the mean change in time to rise from the floor …

Taiho’s pizuglanstat fails in phase III DMD trial

In yet another fail for the Duchenne muscular dystrophy (DMD) field, Taiho Pharmaceutical Co. Ltd.’s pizuglanstat (TAS-205) did not meet the primary endpoint in a phase III trial. The phase III Reach-DMD trial, a randomized, placebo-controlled, double- …

Taiho's late-stage Duchenne candidate fails trial

Taiho Pharmaceutical has suffered a blow to its near-term pipeline after a drug candidate for Duchenne muscular dystrophy (DMD) missed the mark in a phase 3 trial. The REACH-DMD study of pizuglanstat (TAS-205) – a selective haematopoietic prostaglandin D …

Jeffrey Chamberlain, PhD, on the Role of Gene Therapy for Patients with Duchenne Muscular Dystrophy

This interview originally appeared on our sister site, NeurologyLive®. “The idea arises that if you could restore levels of dystrophin up to 15% or maybe even a little higher through a gene therapy approach, that could have really a tremendous impact on …

Palantir Co-Founder Joe Lonsdale Wants A FDA Overhaul With Special Forces-Style AI Team Because China Is Racing To ...

On Monday, Palantir Technologies Inc. PLTR co-founder Joe Lonsdale urged a tech-driven transformation of the Food and Drug Administration, warning that bureaucratic inertia is putting the U.S. behind China in the biotech race. What Happened: In a blog post …

‘Hiking for hope’: Birmingham team climb Snowdon in memory of brother and friend

On Saturday 21 June, Ibrahim Hussain, 22, from Birmingham set off to take on the challenge of climbing Snowdon (Yr Wyddfa), the highest mountain in Wales. He was joined by his two older brothers, Tamir Hussain and Mohammed Nameer, and three friends, and …

Edgewise Therapeutics Reports Positive Results for Muscular Dystrophy Treatment

Edgewise Therapeutics Inc. (NASDAQ:EWTX) is one of the best up and coming stocks to invest in now. On June 26, Edgewise Therapeutics announced positive results from its sevasemten program for Becker and Duchenne muscular dystrophies. For Becker muscular …

Science Day 2025 Zeroes in on Cell and Gene Therapies

Newswise — On June 18, more than 400 researchers, clinicians, educators, and community supporters gathered for Science Day 2025—The Saban Research Institute’s (TSRI) annual symposium and poster session. Attendees heard from experts across the scientific …

Salanersen Shows Promise in Phase 1, MESA Open-Label Data Released, Fenfluramine Meets End Points in Phase 3 GEMZ Trial

WATCH TIME: 4 minutes Welcome to this special edition of Neurology News Network. I'm Marco Meglio. Newly announced interim data from a phase 1 study showed that treatment with salanersen (Biogen), an investigational antisense oligonucleotide …

The online world comes with risks – but also friendships and independence for young people with disabilities

“In the real world, I’m a coward. When I’m online, I’m a hero.” These words, paraphrased from a conversation with a young man with autism, have stayed with us throughout the years of research that underpin our recently published book exploring the …

Dad of tragic Scots boy in government help call after selling home amid treatment nightmare

The dad of a tragic Scots boy has called for more help for families with terminally ill children - after selling their home while shelling out thousands of pounds on his treatment. Devoted dad Jamie Tierney believes there should be a body set up to help …

Voices: We’ve seen the impact of Utah’s groundbreaking genetic research. Federal funding cuts will be devastating for ...

(Francisco Kjolseth | The Salt Lake Tribune) A mass spectrometer at the University of Utah processes coral materials on Thursday, June 9, 2022, to be analyzed in the first step towards determining its usefulness in treating cancer. A team of researchers …

‘The same way Alcoholics Anonymous and NA were there for me, I want the running clubs to be there for others’

Dubliner Chris Connolly on his struggles with addiction, how running saved him and why he set up running clubs that would be there for others who needed a safe space. Next is Ireland’s first autism accredited running club Chris Connolly: ‘Running helped me …

Wave Life Sciences: Speculative, But Promising

Wave Life Sciences could offer significant upside if obesity candidate WVE-007 delivers strong human data, with a large market opportunity and muscle-sparing potential. WVE-N531 for Duchenne muscular dystrophy shows potential best-in-class data and is on …

Dad of Scottish boy with terminal illness forced to sell family home amid call for help

The parents of a Scottish boy with a rare condition have sold their home while shelling out thousands of pounds on his treatment. Six-year-old Jamie Tierney suffers from rare muscle wasting disease Duchenne muscular dystrophy (DMD). His dad, also Jamie, …

Forward Healthy Lifestyles Introduces Revolutionary HIFU Korean Skincare Treatment

GERMANTOWN, WI, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Forward Healthy Lifestyles is proud to announce the launch of its latest innovation in non-invasive skincare: the HIFU (High-Intensity Focused Ultrasound) Korean Skincare Treatment …

Pace Palm Beach Awarded $3,700 Grant from Mary’s Memorial Fund to Support Launch of Psychoeducation Groups Program

Mary’s Memorial Fund Grant will Help Pace Palm Beach Expand Access to Mental Health Programs for Girls PALM SPRINGS, FL, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Pace Center for Girls Palm Beach recently received a generous $3,700 grant …

Onyx and Itiliti Health Launch OnyxOS Connector Solution to Revolutionize Electronic Prior Authorization

Onyx and Itiliti Health launch OnyxOS Connector enabling health plans to comply with CMS Prior Authorization API compliance requirements. MISSION HILLS, KS, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Onyx, a leading innovator in …

GRX Marketing Celebrates 15 Years of Trusted Pharmacy Marketing Solutions

Trusted marketing agency marks milestone with ongoing innovation and dedication to independent pharmacy success. DES MOINES, IA, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- GRX Marketing, a trusted leader in pharmacy marketing solutions, proudly …

La Jolla Vein & Vascular Earns Third Consecutive IAC Reaccreditation, Solidifying Leadership in Advanced Vein Care

Commitment to Accreditation, Patient Safety, and Personalized Care Solidifies La Jolla Vein & Vascular as Southern California’s Premier Vascular Care Leader LA JOLLA, CA, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- La Jolla Vein & …

Dr. Mark Hawass selected as Top Doctor of the Year in Regenerative and Functional Medicine by IAOTP

Dr. Mark Hawass honored member of the International Association of Top Professionals (IAOTP) will be honored at their annual awards gala in Las Vegas NEW YORK, NY, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Dr. Mark Hawass, a highly esteemed …

Empowering Parents KC Receives 2025 Best of Lee's Summit Award

Empowering Parents KC earns 2025 Best of Lee’s Summit Award in Mental Health Services for the third year in a row. KANSAS CITY, MO, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Empowering Parents KC (EPKC) has once again been recognized for …

New Report: Despite Leading in Medical Education, New York Ranks 49th in Hospital Access

A new study reveals that despite world-class medical schools, many states face a dire shortage of accessible hospitals and staff. LOS ANGELES, CA, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- - New York ranks 49th nationally for hospital …

Blue Goat Cyber Leads Medical Device Cybersecurity Compliance as FDA Finalizes New Guidance

Blue Goat Cyber leads medical device cybersecurity compliance, helping manufacturers meet the FDA’s finalized cybersecurity guidance issued June 27, 2025. SCOTTSDALE, AZ, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- On June 27, 2025, the U. …

The Nu-Age Group Expands Services With Coresite Data Center Solutions

**The Nu-Age Group Partners with Coresite to Deliver Compliant, Scalable, and Secure Data Center Solutions Nationwide** SECAUCUS, NJ, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- The Nu-Age Group, a leading provider of nationwide Managed IT …

FIGUR8 Launches Recovery Indicator, Advancing Musculoskeletal Care Through AI-Driven Recovery Intelligence

BOSTON, MA, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- FIGUR8, the market leader for measuring musculoskeletal (MSK) health and injury recovery, today announced the launch of the FIGUR8 Recovery Indicator, a breakthrough feature that …

Healing and Empowerment Practitioner Dr. Jennifer Nestell Recently Featured on Close Up Radio

BERLIN, NH, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Jennifer Nestell, PhD, an experienced clinician dedicated to healing through depth psychology and trauma recovery, is proud to announce her innovative tele-health practice addressing …

Q2 2025 Digital Health Funding Report: Investors Back AI With Purpose as Deal Size Rebounds

AI-powered ventures lead the way as digital health funding hits $12.1B in Q2 2025, with Europe outpacing global trends and deal size rebounding. BOSTON, MA, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- AI Drives Bigger Bets as Europe Surges …

Fayetteville Dentist Scott Bell, DDS Launches New Website Showcasing Comprehensive Dental Care

The new website for Bell Dental Cosmetic & Family introduces an educational, patient-focused platform highlighting the practice’s full range of services. FAYETTEVILLE, AR, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Dr. Scott Bell, a dentist …

Avaamo Ambient announces Integration with Sunrise Clinical Manager (SCM)

Addressing Clinician Burnout, Avaamo Ambient Delivers Context-Aware AI to Improve Documentation Accuracy and Workflow Seamlessness in SCM LOS ALTOS, CA, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Avaamo today announced the launch of its …

Wellness Retreat 'Zenbo Seinei' Introduces New 'Evening Breeze ZEN Tea Plan'

The limited-time zen meditation and Japanese tea summer plan continues through Sunday, August 31 AWAJI CITY, HYOGO PREFECTURE, JAPAN, July 9, 2025 /⁨EINPresswire.com⁩/ -- Zenbo Seinei, a zen retreat facility located on Awaji Island, Hyogo Prefecture, has …

Plantation Villa Celebrates the Success of Its 200-Hour Yoga Teacher Training

Plantation Villa, Sri Lanka’s renowned wellness retreat centre, proudly reflects on the success of its 200-hour Yoga Teacher Training (YTT) held in June. HEREFORD, KINGS CAPLE, UNITED KINGDOM, July 9, 2025 /⁨EINPresswire.com⁩/ -- Plantation Villa, …

Diversified Botanics Welcomes Dr. Robert R. Redfield Jr. to Board of Directors Mission to Combat Emerging Opioid Crisis

BALTIMORE, MD, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Diversified Botanics, a pioneering science-based firm at the forefront of botanical health and wellness, is thrilled to announce that Dr. Robert R. Redfield Jr., renowned virologist …

AVERARDO ORTA (COLIBRI' GROUP), SAYS AI IS TRANSFORMING WORLD OF HEALTHCARE ON THE PLANET

"The introduction of AI in imaging has already demonstrated extraordinary results," says Averardo Orta. BOLOGNA, BOLOGNA, ITALY, July 9, 2025 /⁨EINPresswire.com⁩/ -- Artificial intelligence is rapidly transforming the global healthcare …

A Vintage Vision with a Modern Mission: Pin-Ups For Vets Unveils 20-Year Anniversary Calendar Featuring Female Veterans

The 2026 Pin‑Ups For Vets calendar blends nostalgic 1940s glamour with today's female Veterans to reimagine them as pin-up icons and to celebrate their service. CLAREMONT, CA, UNITED STATES, July 9, 2025 /⁨EINPresswire.com⁩/ -- Pin-Ups For …