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Published on 11:00 GMT

Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201, a Phase 1/2 Multiple ...

– Company on track to initiate ELEVATE-45-201 study in Q3 2025 – – ELEVATE-45 regulatory filings submitted in the EU, with regulatory review ongoing – – ELEVATE-45 is the second of three novel exon skipping Duchenne programs the Company expects to …

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Published on 17:36 GMT

Phase 2 Trial Data Shows Friedreich Ataxia Therapy Nomlabofusp Increases Frataxin Levels

New phase 2 clinical trial (NCT05579691) data showed that nomlabofusp (Larimar Therapeutics), an invesigational therapy for Friedreich ataxia (FA), was well tolerated and significantly increased frataxin levels in treated patients. Presented at the 2025 …

Source: NeurologyLive - Neutral
Published on Mar 23, 2025

MDA Study Highlights Lack of Psychosocial Care Services for Duchenne Muscular Dystrophy

Findings from a single-center clinic study showcased the lack of psychosocial services among patients with Duchenne muscular dystrophy (DMD), as only one-fourth of observed patients had access to these necessary care functions. Overall, these findings …

Source: NeurologyLive - Neutral
Published on 23:12 GMT

StemGenex Biologic Laboratories, LLC - 557907 - 10/31/2018

WARNING LETTER UNITED PARCEL SERVICE SIGNATURE REQUIRED October 31, 2018 Warning Letter #OBPO - 19-02 Rita F. Alexander, Owner/Manager Jenny R. Galloway, MD, Laboratory and Medical Director StemGenex Biologic Laboratories, LLC 11515 El Camino Real, …

Distribution channels: Healthcare & Pharmaceuticals Industry
Published on 13:29 GMT

Short-Term Analysis Confirms Safety of Oral Edaravone in ALS Over 96 Weeks

Mitsubishi Tanabe Pharma America’s (MTPA) oral formulation of edaravone (Radicava ORS) demonstrated a consistent safety profile over 96 weeks in patients with amyotrophic lateral sclerosis (ALS), according to findings from the phase 3 MT-1186-A03 study ( …

Source: NeurologyLive - Label Pending
Published on 13:24 GMT

Augustine decloaks with $85m for nerve disease programme

Augustine Therapeutics has raised €78 million ($85 million) in first-round funding for a pipeline headed by a candidate for the inherited neurological disorder Charcot-Marie-Tooth (CMT). The Leven, Belgium-based biotech has built its business around a …

Source: pharmaphorum - Neutral
Published on Mar 22, 2025

Sevasemten Reduces Muscle Injury Biomarkers of Becker Muscular Dystrophy in Latest Analysis

An analysis of the phase 2 CANYON trial (NCT05291091) showed that treatment with 10 mg of investigational sevasemten (Edgewise Therapeutics) led to reductions in notable muscle injury biomarkers in adults with Becker muscular dystrophy (BMD). Overall, the …

Source: NeurologyLive - Neutral
Published on Mar 23, 2025

Gene editing may be key to treating head and neck cancers

Researchers in Israel have found a way to use revolutionary gene-editing technology to treat head and neck cancers. They say they can identify the single gene that fuels the growth of the cancer cells – and remove it. The team at Tel Aviv University uses …

Source: Israel21C - Uncategorized
Published on Mar 20, 2025

Gene Therapy RGX-202 Shows Positive Interim Results in Duchenne Muscular Dystrophy Trial

New, positive interim data from the phase 1/2 AFFINITY DUCHENNE trial (NCT05693142) of RGX-202 (REGENXBIO), an investigational gene therapy for Duchenne muscular dystrophy (Duchenne), were recently presented at the 2025 Muscular Dystrophy Association ( …

Source: NeurologyLive - Neutral
Published on 19:14 GMT

12 Most Promising Biotech Stocks According to Wall Street Analysts

In this article, we will be taking a look at the 12 most promising biotech stocks according to Wall Street analysts. The biotechnology sector is gaining new momentum with better market conditions, cutting-edge innovations, and growing investor attention. …

Source: Insider Monkey - Neutral
Published on 21:51 GMT

MDA Reflects on 75 Years of Research and the Precarious Times Ahead

Neuromuscular disease researchers, clinicians, and advocates gathered to celebrate 75 years of progress at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Dallas, Texas, but they also noted that these gains could be …

Source: The American Journal of Managed Care - Neutral
Published on 21:51 GMT

Building Community and Advancing Care at MDA 2025

The 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference held in Dallas, Texas, marked the 75th anniversary of the MDA. As clinicians harkened back to the most valuable elements of the meeting, they highlighted the great …

Source: The American Journal of Managed Care - Neutral
Published on Mar 21, 2025

Combining EZH2 inhibitors boosts treatment effects for Duchenne muscular dystrophy

A Korean research team has proposed a new treatment strategy that could improve treatment effectiveness for Duchenne muscular dystrophy (DMD), a rare inherited disease for which steroid treatment is the standard of care. The team further confirmed that the …

Source: Korea Biomedical Review - Neutral
Published on Mar 20, 2025

Givinostat Delays Duchenne Muscular Dystrophy Progression by 2 Years, Simulation Model Shows

Givinostat (Duvyzat; Italfarmaco SpA), an oral histone deacetylase inhibitor recently approved by the FDA for Duchenne muscular dystrophy (DMD) in patients aged 6 years and older, was associated with a significant delay in disease progression, according to …

Source: NeurologyLive - Label Pending
Published on Mar 19, 2025

SHIELD-DMD to Evaluate Therapeutic Potential of IL-6 Receptor Satralizumab in Duchenne Muscular Dystrophy

A new phase 2 clinical trial, dubbed SHIELD-DMD (NCT06450639), is evaluating satralizumab (Enspryng; Roche), a monoclonal antibody targeting the interleukin-6 receptor (IL-6R), for its potential to improve bone strength and muscle function in boys with …

Source: NeurologyLive - Neutral
Published on Mar 21, 2025

REGENXBIO’s DMD Gene Therapy RGX-202 Generates Microdystrophin Expression in Additional Patients Treated in Phase 1/2 ...

REGENXBIO’s RGX-202, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Duchenne muscular dystrophy (DMD), has continued to demonstrate the ability to produce microdystrophin at “consistent, robust” levels in data …

Source: CGTLive - Neutral
Published on 07:21 GMT

CGT sector: Cautious optimism despite numerous headwinds

In the first quarter this year, there was only one US approval in the CGT space – Neurotech’s Encelto (revakinagene taroretcel). And according to US Food and Drug Administration (FDA) approval target dates for the rest of the year, only one cell therapy …

Source: BioProcess International - Neutral
Published on Mar 21, 2025

FDA Approves Vutrisiran for ATTR-CM, Expanding Indication in Amyloidosis

The FDA has approved vutrisiran (Amvuttra; Alnylam Pharmaceuticals) for the treatment of cardiomyopathy associated with wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults.1 This expanded indication makes vutrisiran both the …

Source: NeurologyLive - Label Pending
Published on Mar 21, 2025

Investors shift focus from gene therapy to lucrative weight-loss drugs

Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward medical sectors like obesity and cancer, as sales for some of the new treatments fall short. In the last year, some drugmakers have …

Source: The News International - Uncategorized

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Published on March 24, 2025 - 09:18 GMT

Octal’s Generative AI Chatbots Reshape Healthcare Delivery with Advanced Software

Octal IT Solution is transforming healthcare with its AI chatbots, improving patient care and operational efficiency. SARASOTA, FL, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- The healthcare industry is rapidly embracing …

Distribution channels: Business & Economy, Companies ...
Published on March 24, 2025 - 12:33 GMT

Westborough Behavioral Healthcare Hospital Welcomes CEO Dr. Shawn Daugherty

A new era of growth, innovation, and patient-centered care begins at Westborough Behavioral Healthcare Hospital WESTBOROUGH, MA, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- Westborough Behavioral Healthcare Hospital (WBHH) is pleased to …

Distribution channels: Healthcare & Pharmaceuticals Industry ...

Published on March 24, 2025 - 20:28 GMT

World Estimating regulates its healthcare standards once more

World Estimating Services reviews its healthcare standards and work policies for employee NEW YORK, NY, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- We are familiar with the proverb “All work and no play make Jack a dull boy”. This …

Distribution channels: Building & Construction Industry ...
Published on March 24, 2025 - 18:43 GMT

Rethinking Medicine: Dr. Michael Borger’s Bold Challenge to the Healthcare System

Discover a bold new perspective on healthcare in Self-Referral/Maximum Health by Dr. Michael Borger. DALLAS, TX, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- For decades, the healthcare system has been fighting a war against disease. …

Distribution channels: Book Publishing Industry ...

Published on March 24, 2025 - 19:27 GMT

Dr. Robert Corkern Announces Grant for Healthcare Students Addressing Economic Challenges in the Industry

GREENVILLE, Miss., March 24, 2025 (GLOBE NEWSWIRE) -- The Dr. Robert Corkern Grant for Healthcare Students is now accepting applications for the 2025 award. This prestigious grant, founded by Dr. Robert Corkern, provides a $1,000 scholarship to an …

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Published on March 24, 2025 - 19:12 GMT

Dr. Fazal Panezai Grant for Healthcare Students Announces Award to Support Future Medical Leaders

MATAWAN, N.J., March 24, 2025 (GLOBE NEWSWIRE) -- The Dr. Fazal Panezai Grant for Healthcare Students is now accepting applications from undergraduate students pursuing careers in healthcare. Founded by Dr. Fazal Panezai, a distinguished physician with …

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Published on March 24, 2025 - 16:30 GMT

Better Choice Announces SRx Health’s Participation at the 2025 Bloom Burton & Co. Healthcare Investor Conference on May ...

TAMPA, Fla., March 24, 2025 (GLOBE NEWSWIRE) -- Better Choice Company (NYSE: BTTR) (“Better Choice” or “the Company”), a pet health and wellness company, today announced that SRx Health’s management team will be presenting at the 2025 Bloom Burton & Co …

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Published on March 24, 2025 - 14:26 GMT

Guggenheim Securities Expands Healthcare Investment Banking Practice With the Addition of Paul Hepper

NEW YORK, March 24, 2025 (GLOBE NEWSWIRE) -- Guggenheim Securities, the investment banking and capital markets division of Guggenheim Partners, announced today that Paul Hepper will join the firm’s Healthcare investment banking practice to focus on …

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Published on March 24, 2025 - 23:15 GMT

Helvetic Dental Clinics en Hongrie célèbre une année record en 2024 en tant que meilleure clinique dentaire de Budapest

Helvetic Dental Clinics, la meilleure clinique dentaire en Hongrie, termine 2024 sur un bilan record et agrandit sa capacité avec l’ajout de quatre fauteuils. BUDAPEST, BUDAPEST, HUNGARY, March 25, 2025 /⁨EINPresswire.com⁩/ -- Helvetic Dental …

Distribution channels: Beauty & Hair Care, Business & Economy ...
Published on March 24, 2025 - 23:12 GMT

Chasin A Dream Foundation’s Annual Gala Raises Over $550,000 for Local Children Battling Life-Threatening Illnesses

The Jupiter based foundation's annual gala was an unforgettable evening of generosity and impact, breaking a record with $550,000 raised. JUPITER, FL, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- Chasin A Dream Foundation’s annual …

Distribution channels: Conferences & Trade Fairs, Culture, Society & Lifestyle ...
Published on March 24, 2025 - 22:57 GMT

Study Validates Effectiveness of KYAN Tech's Platform for Combination Therapy Prediction and Target Discovery in Sarcoma

Groundbreaking study published in npj Precision Oncology highlights Optim.AI’s potential to revolutionize personalized cancer treatment strategies SINGAPORE, SINGAPORE, March 24, 2025 /⁨EINPresswire.com⁩/ -- KYAN Technologies Pte. Ltd. (“KYAN”) …

Distribution channels: Companies, Education ...
Published on March 24, 2025 - 21:13 GMT

ANJC Hosts Successful Marketing Workshop Featuring Saleem Musallam of Excite Medical in Clark, NJ

Chiropractors gained valuable guidance in spinal decompression marketing at a sold-out ANJC workshop, while Excite Medical announces upcoming events. CLARK, NJ, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- The Association of New Jersey …

Distribution channels: Healthcare & Pharmaceuticals Industry ...
Published on March 24, 2025 - 21:07 GMT

Spine and Joint Center Celebrates 45+ Years of Chiropractic Excellence in Fort Lauderdale

Providing expert chiropractic care for over 45 years, Spine and Joint Center in Fort Lauderdale offers personalized pain relief and wellness treatments. FORT LAUDEDALE, FL, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- For 45 years, Spine …

Distribution channels: Healthcare & Pharmaceuticals Industry ...
Published on March 24, 2025 - 19:51 GMT

High Fashion Hits the Runway at Westport Rehabilitation and Nursing Center

High Fashion Hits the Runway at Westport Rehabilitation and Nursing Center MARLTON, NJ, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- High Fashion Hits the Runway at Westport Rehabilitation and Nursing Center Residents of Westport …

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Published on March 24, 2025 - 18:57 GMT

Health, Wellness, and Longevity Event: Screening of PBS Documentary 'The [M] Factor'

ORMOND BEACH, FL, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- ZIA Health and Enchanted Medical Aesthetics are thrilled to announce an enlightening evening dedicated to health, wellness, and longevity. Join us for a special screening of …

Distribution channels: Culture, Society & Lifestyle, Healthcare & Pharmaceuticals Industry ...
Published on March 24, 2025 - 18:14 GMT

SkinGenuity Debuts Groundbreaking Product Collection at AMWC Monaco

LARGO, FL, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- SkinGenuity, LLC, announced today it will showcase its innovative collection of products for skin, hair, lashes and women’s health at the upcoming Aesthetic and Anti-Aging Medicine World …

Distribution channels: Beauty & Hair Care, Conferences & Trade Fairs ...
Published on March 24, 2025 - 18:00 GMT

Conductor and Educator Dr. Jason Noble Announces Upcoming Engagements

Noble kicks off slate of performances after recent brain surgery NEW YORK, NY, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- Dr. Jason Noble, conductor and educator whose PRISM Arts Education Research Initiative transforms how we …

Distribution channels: Business & Economy, Culture, Society & Lifestyle ...
Published on March 24, 2025 - 17:43 GMT

Ayurvedic & Naturopathic Medical Clinic Leads the Way in Integrative Oncology and Ayurvedic Medicine for the Modern Era

Ayurvedic & Naturopathic Medical Clinic blends ancient wisdom with modern science to deliver personalized, integrative cancer care. BELLEVUE, WA, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- As interest in whole-body, patient-centered care …

Distribution channels: Culture, Society & Lifestyle, Healthcare & Pharmaceuticals Industry ...
Published on March 24, 2025 - 17:19 GMT

Lumenia Consulting to celebrate 10th UK ERP HEADtoHEAD™ event

The 10th UK Lumenia Consulting ERP HEADtoHEAD™ event will take place on 2-3 April in Milton Keynes. Compare leading ERP solutions at one event. LONDON, BUCKINGHAMSHIRE, UNITED KINGDOM, March 24, 2025 /⁨EINPresswire.com⁩/ -- The 10th UK Lumenia …

Distribution channels: Aviation & Aerospace Industry, Building & Construction Industry ...
Published on March 24, 2025 - 16:32 GMT

Executive, Talent Strategist Sonji Young Guides Jobseekers To New Opportunities

According to Reuters, the US job market appears to be steady, though job opportunities are limited. Talent Strategist, Sonji Young has advice for job seekers. BEAVERTON, OR, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- With the US job …

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Published on March 24, 2025 - 16:21 GMT

HOHLI – Healthy Oceans, Healthy Lives

The University of Basilicata and the Municipality of Pollica launch HOHLI, exploring links between health, fertility, oceans, and the Mediterranean Diet POLLICA, SALERNO, ITALY, March 24, 2025 /⁨EINPresswire.com⁩/ -- The HOHLI – Healthy Oceans, …

Distribution channels: Healthcare & Pharmaceuticals Industry ...
Published on March 24, 2025 - 16:04 GMT

Whittington, Garrison, and Campbell: Challenges With Judging and Interpreting a Drug’s Launch Price (AJMC Commentary)

A new publication suggests that decision makers take both a product lifetime view and a broader societal perspective when judging and rewarding value creation. WASHINGTON, DC, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- Drug prices at …

Distribution channels: Business & Economy, Healthcare & Pharmaceuticals Industry ...
Published on March 24, 2025 - 16:00 GMT

Creative Biolabs Exosomal Small RNA/miRNA Sequencing: Progressing in Biomarkers of Disease Detection

Creative Biolabs provides comprehensive services in exosome small RNA sequencing including exosome isolation, miRNA extraction, sequencing, and data processing. SHIRLEY, NY, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- As important …

Distribution channels: Healthcare & Pharmaceuticals Industry, Science ...
Published on March 24, 2025 - 15:52 GMT

ALSO Youth Raises $35,530 at Sold-Out Tsunami Fundraiser to Support LGBTQ+ Youth in Sarasota & Manatee

ALSO Youth’s sold-out brunch fundraiser raised $35,530 to fund free LGBTQ+ youth support programs and mental health services in Sarasota and Manatee Counties. SARASOTA, FL, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- ALSO Youth hosted a …

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Published on March 24, 2025 - 15:16 GMT

Pictor Labs & Pramana Announce Strategic Partnership to Revolutionize Digital Pathology with On-Demand Virtual Staining

LOS ANGELES, CA, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- Pictor Labs, a pioneer in AI-driven digital pathology solutions, and Pramana, a leader in next-generation whole-slide digital pathology scanning solutions, today announced a …

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Published on March 24, 2025 - 15:15 GMT

LifeScienceHistory.com Releases Cartoon Illustrating “Milestones in Fluoride”

Learn about the historical milestones in science behind this misunderstood natural mineral. SEATTLE, WA, UNITED STATES, March 24, 2025 /⁨EINPresswire.com⁩/ -- LifeScienceHistory.com, announced the release of its newest cartoon entitled: “ …

Distribution channels: Education, Healthcare & Pharmaceuticals Industry ...
Published on March 24, 2025 - 15:12 GMT

Global Cerebral Adrenoleukodystrophy (CALD) Market Outlook 2025-2034: Growth Drivers, Share, And Trends

The Business Research Company's Cerebral Adrenoleukodystrophy (CALD) Global Market Report 2025 – Market Size, Trends, And Global Forecast 2025-2034 LONDON, GREATER LONDON, UNITED KINGDOM, March 24, 2025 /⁨EINPresswire.com⁩/ -- The focal point …

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Published on March 24, 2025 - 15:11 GMT

Anakinra Market Analysis: Key Trends, Share, Growth Drivers, And Forecast 2025-2034

Anakinra Global Market Report 2025 – Market Size, Trends, And Global Forecast 2025-2034 LONDON, GREATER LONDON, UNITED KINGDOM, March 24, 2025 /⁨EINPresswire.com⁩/ -- Updated 2025 Market Reports Released: Trends, Forecasts to 2034 – Early Purchase …

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