Phenylketonuria Drugs News Monitoring

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Published on Mar 24, 2025

Newborn Screening Uncovers Hidden Genetic Disorders

Newswise — How can a seemingly healthy newborn have a genetic disorder? Kimberly Chapman, MD, PhD, Section Chief, Medical Genetics at Children’s Hospital Los Angeles explains how newborn screening works, and how it can prevent lifelong disability—and …

Source: Newswise - Neutral
Published on Mar 20, 2025

Phenylketonuria Treatment Market Set to Surge at 7.8% CAGR, to Reach US$ 1.62 Billion by 2034: Fact.MR Report

Awareness Campaigns by Governments and Regulatory Bodies Regarding Phenylketonuria Treatment Playing Key Role in Market Expansion: Fact.MR Report ROCKVILLE, MD, UNITED STATES, March 20, 2025 /⁨EINPresswire.com⁩/ -- According to a recently updated …

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Published on Mar 20, 2025

PTC Therapeutics Reveals New Sepiapterin Data From Phase 3 Study In Patients With Inherited Disorder, FDA Decision ...

On Thursday, PTC Therapeutics, Inc. PTCT shared new data from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. These data provide …

Source: Benzinga - Neutral
Published on 09:06 GMT

Prevalence and Demographic Analysis of Hemoglobinopathies in Newborns: A Three-Year Study at Thumbay Teaching Hospital, ...

Introduction Newborn Screening (NBS) Program For almost four decades, Newborn screening (NBS) has been a major part for the prevention of hemoglobinopathies such as Sickle cell disease (SCD), Thalassemia and other abnormal hemoglobin which aim in avoiding …

Source: Dove Medical Press - Neutral
Published on Mar 20, 2025

The Expression of Ferroptosis-Related Genes in Hepatocellular Carcinoma and Their Relationships With Prognosis

Introduction Global Cancer Statistics 2024 shows that primary liver cancer is the 12th most common cancer in the world in 2024, with the sixth highest mortality rate, and it is an extremely malignant tumor.1 Among them, hepatocellular carcinoma (HCC) …

Source: Dove Medical Press - Neutral
Published on Mar 20, 2025

Association Between Social Capital and Anxiety Among Older Adults in China: A Cross-Sectional Study

1School of Nursing, XiangNan University, Chenzhou, People’s Republic of China; 2College of Nursing and Allied Health Sciences, St. Paul University Manila, Manila, Philippine; 3School of Chemical and Environmental Sciences, XiangNan University, Chenzhou, …

Source: Dove Medical Press - Neutral

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Published on March 25, 2025 - 11:30 GMT

Quoin Pharmaceuticals Files U.S. Patent Application for Novel Topical Formulations to Treat Netherton Syndrome and Other ...

Target Indications Also Include Peeling Skin Syndrome, SAM Syndrome, Palmoplantar Keratoderma and Severe Atopic Dermatitis Third Quoin Patent Application for Netherton Syndrome If Granted Company Would have Patent Protection for QRX003 for Netherton …

Distribution channels: Healthcare & Pharmaceuticals Industry, Law ...
Published on March 25, 2025 - 11:30 GMT

Traws Pharma’s COVID-19 Candidate, Ratutrelvir, Presented at ICAR

Preclinical and Phase 1 data suggest that ratutrelvir can be used without ritonavir and may reduce the likelihood of COVID rebound and the risk of long COVID due to a longer treatment regimen Preparations are underway for FDA interactions and initiation of …

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Published on March 25, 2025 - 11:30 GMT

Relmada Therapeutics Licenses Phase 2 Bladder Cancer Candidate, NDV-01, from Trigone Pharma, Ltd.

NDV-01 is a novel, sustained-release, intravesical gemcitabine/docetaxel, ready-for-use product candidate for the treatment of non-muscle invasive bladder cancer (NMIBC, U.S. prevalence of ~600,000 patients, with ~62,000 newly diagnosed patients …

Distribution channels: Companies, Technology ...
Published on March 25, 2025 - 11:00 GMT

NMD Pharma presents preclinical data highlighting increased innervation of skeletal muscle with ClC-1 ion channel ...

Company presented clinical and preclinical data at the MDA Conference last week in Dallas, Texas Oral presentation included data from MuSK-MG rat model depicting increases in muscle function and improvements in the structural integrity of the neuromuscular …

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Published on March 25, 2025 - 10:01 GMT

X4 Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Corporate Update

Phase 3 4WARD trial in chronic neutropenia now activated at ~90% of targeted trial sites; full enrollment expected in 3Q or 4Q 2025; top-line data anticipated in 2H 2026 4WARD trial protocol refined, increasing confidence in successful outcome 2024 …

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Published on March 25, 2025 - 06:30 GMT

Alumis and Kaken Pharmaceutical Announce Collaboration and Licensing Agreement for ESK-001 in Dermatology in Japan

- Alumis to receive $40 million in upfront and near-term co-development payments, with potential for approximately $140 million in additional milestone and field option payments, plus tiered royalties on future sales -Deal underscores the commercial …

Distribution channels: Technology ...
Published on March 25, 2025 - 06:00 GMT

NMD Pharma presents preclinical data highlighting increased innervation of skeletal muscle with ClC-1 ion channel ...

Company presented clinical and preclinical data at the MDA Conference last week in Dallas, Texas Oral presentation included data from MuSK-MG rat model depicting increases in muscle function and improvements in the structural integrity of the neuromuscular …

Distribution channels: Media, Advertising & PR, Technology ...
Published on March 25, 2025 - 03:13 GMT

Lawsuit Notice: Investors who hold Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) shares should contact the Shareholders ...

SAN DIEGO, March 24, 2025 (GLOBE NEWSWIRE) -- Shareholders Foundation, Inc. announced that a lawsuit is pending for certain investors in shares of Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN). Investors who purchased shares of Regeneron Pharmaceuticals, …

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Published on March 25, 2025 - 11:30 GMT

Mobility City's Technician Network, the Largest HME Repair & Delivery Network, is Now an Accredited Exemplary Provider

Mobility City's franchise system, with the largest HME repair and delivery Technician Network in the US, is now a CMS approved Exemplary Provider BOCA RATON, FL, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- Mobility City Holdings Inc, …

Distribution channels: Companies, Healthcare & Pharmaceuticals Industry ...
Published on March 25, 2025 - 11:30 GMT

Foreign Aid Bridge Fund Donations Surpass $1 Million

Emergency Funding Fuels Life-Saving Relief for Frontline Organizations Amid Rising Global Crises WASHINGTON, RI, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- The Foreign Aid Bridge Fund has surpassed $1 million in donations just over a …

Distribution channels: Emergency Services, Healthcare & Pharmaceuticals Industry ...
Published on March 25, 2025 - 11:00 GMT

Enkrypt AI Launches New Multimodal AI Security Solution to Safeguard Text, Image, and Voice AI Systems

Innovative solution secures multimodal AI systems from emerging security, bias, and compliance risks BOSTON, MA, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- Enkrypt AI, a leader in AI security and compliance – is proud to announce the …

Distribution channels: Banking, Finance & Investment Industry, Business & Economy ...
Published on March 25, 2025 - 11:00 GMT

CMTA’s $300K Investment in CRISPR for CMT2A

CMTA invests $300K in CRISPR gene editing for CMT2A at UCSF’s Gladstone Institutes, targeting MFN2 mutations to restore nerve function. GLENOLDEN, PA, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- The Charcot-Marie-Tooth Association (CMTA …

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Published on March 25, 2025 - 11:00 GMT

Dean Melissa Perry appointed to prestigious National Academies of Sciences, Engineering, and Medicine Committee

Perry appointed to NASEM Committee on Clinical Follow-Up and Care for Those Impacted by the JP-5 Releases at Red Hill. FAIRFAX, VA, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- As a widely respected researcher and public health leader, …

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Published on March 25, 2025 - 11:00 GMT

LarmorBio Announces Addition of Multiple New Board Members and Senior Executive Hires

Company Prepares to Scale Business with Proprietary Microscale Magnetic Resonance Technology BOSTON, MA, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- LarmorBio, a Massachusetts Institute of Technology (MIT) start-up pioneering the use of …

Distribution channels: Healthcare & Pharmaceuticals Industry ...
Published on March 25, 2025 - 10:45 GMT

Feroot Launches PaymentGuard AI: Automated PCI DSS 4 Compliance for Websites with E-commerce Payment Webpages.

PaymentGuard AI by Feroot Security: Automating PCI DSS 4 Compliance with Requirements 6.4.3 and 11.6.1 for SAQ D and SAQ A-EP E-Commerce Merchants. TORONTO, CANADA, March 25, 2025 /⁨EINPresswire.com⁩/ -- Feroot Security, a leader in cybersecurity …

Distribution channels: Banking, Finance & Investment Industry, Healthcare & Pharmaceuticals Industry ...
Published on March 25, 2025 - 10:45 GMT

PUSH for Empowered Pregnancy Launches #MomentForStillness to Honor Babies Born Still and Demand Urgent Action

A Nationwide Call to Address Preventable Stillbirths Begins March 25th at Noon ET NEW YORK, NY, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- PUSH for Empowered Pregnancy is launching the #MomentForStillness campaign at noon ET on Tuesday, …

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Published on March 25, 2025 - 10:18 GMT

TravelingWiki Foundation Launches 'Card For a Cause' to Leverage Sports Cards for Free Non Visible Disability Resources

TravelingWiki Now Offers Free Resources in English, Spanish, French, German, Mandarin, Japanese, Hebrew, Arabic, Portuguese, Hindi, Twi, Vietnamese & Russian DES MOINES, IA, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- TravelingWiki …

Distribution channels: Aviation & Aerospace Industry, Healthcare & Pharmaceuticals Industry ...
Published on March 25, 2025 - 10:00 GMT

PhenoBiome Welcomes New Leadership and Validates Breakthrough Microbiome Modulation

Brian Russon Named CEO, Kait Mikes Appointed COO as Company Advances Clinical Validation of Predictive Platform PALM SPRINGS, CA, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- PhenoBiome, Inc., a pioneering leader in personalized …

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Published on March 25, 2025 - 09:45 GMT

Florida Mesothelioma Victims Center Has Endorsed the Gori Law Firm for a Former Power Plant Worker with Just Diagnosed ...

TAMPA , FL, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- The Florida Mesothelioma Victims Center says, "We have endorsed, and we highly recommend the Gori Law Firm for a former power plant worker with just diagnosed mesothelioma or …

Distribution channels: Automotive Industry, Building & Construction Industry ...
Published on March 25, 2025 - 09:43 GMT

OmnySense to Target Oncology Patients as Initial Focus for Its Transformative Personal Health-Assessment Solution

19m Americans have cancer. Unplanned acute visits drive nearly half the costs-creating a major unmet need and opportunity for innovative solutions TEL AVIV, ISRAEL, March 25, 2025 /⁨EINPresswire.com⁩/ -- OmnySense, an innovative digital health …

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Published on March 25, 2025 - 08:17 GMT

RSU Research Week Opening Session highlights the interaction between medicine, technology, and behavioural science

RSU Research Week 2025 opened on 24 March with an ambitious plenary session focusing on the role of research, technology, and the human factor in healthcare. RIGA, LATVIA, March 25, 2025 /⁨EINPresswire.com⁩/ -- RSU Research Week 2025 opened on 24 …

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Published on March 25, 2025 - 08:09 GMT

Dan Pitulia Appointed as Group CEO of the Atlas Antibodies Group

The Board of Atlas Antibodies Group is pleased to announce the appointment of Dan Pitulia as the new Group Chief Executive Officer (CEO). STOCKHOLM, SWEDEN, March 25, 2025 /⁨EINPresswire.com⁩/ -- The Board of Atlas Antibodies Group is pleased to announce …

Distribution channels: Business & Economy, Chemical Industry ...
Published on March 25, 2025 - 07:36 GMT

A' Wellness, Health Promotion, and Preventive Care Design Award Announces Comprehensive Prize Package for 2024

The prestigious A' Wellness, Health Promotion, and Preventive Care Design Award reveals an extensive array of benefits for 2024 competition laureates COMO, CO, ITALY, March 25, 2025 /⁨EINPresswire.com⁩/ -- The A' Wellness, Health Promotion …

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Published on March 25, 2025 - 05:24 GMT

Unlocking the Potential of Tofacitinib: New Frontiers in Treating Skin Disorders

BRADENTON, FL, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- Skin disease related to immune system problems can be a challenge to treat. A new drug, Tofacitinib has gained attention for its therapeutic effects in autoimmune and inflammatory …

Distribution channels: Beauty & Hair Care, Education ...
Published on March 25, 2025 - 05:08 GMT

Acclaim Autism Opens New Early Childhood ABA Therapy Center in Lancaster, PA

Acclaim Autism Opens New Early Childhood ABA Therapy Center in Lancaster, PA: Empowering young children with autism through individualized therapy. LANCASTER, PA, UNITED STATES, March 25, 2025 /⁨EINPresswire.com⁩/ -- Acclaim Autism is thrilled to …

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Published on March 25, 2025 - 11:30 GMT

Cognition Therapeutics to Report Biomarker Results from Phase 2 SHINE Study in Mild-to-Moderate Alzheimer’s Disease in ...

PURCHASE, N.Y., March 25, 2025 (GLOBE NEWSWIRE) -- Cognition Therapeutics, Inc., (the “Company” or “Cognition”) (NASDAQ: CGTX), a clinical-stage company developing drugs that treat neurodegenerative disorders, will be presenting biomarker results from the …

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Published on March 25, 2025 - 11:30 GMT

Enable Injections Announces Regulatory CE Mark Approval in Europe for enFuse Technology

CINCINNATI, March 25, 2025 (GLOBE NEWSWIRE) -- Enable Injections, Inc. (“Enable”), a healthcare innovation company developing and manufacturing the enFuse® wearable drug delivery platform, today announced it has received its European Union Medical Device …

Distribution channels: Healthcare & Pharmaceuticals Industry, Media, Advertising & PR ...
Published on March 25, 2025 - 11:30 GMT

CorMedix Inc. Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update

‒ Q4 2024 Net Revenue of $31.2mm; 2024 Net Revenue of $43.5mm ‒ ‒ Q4 Net Income of $13.5mm and Adjusted EBITDA of $15.3mm ‒ ‒ Conference Call Scheduled for Today at 8:30 a.m. Eastern Time ‒ BERKELEY HEIGHTS, N.J., March 25, 2025 (GLOBE NEWSWIRE) -- …

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