PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Composite endpoints might include factors such as hospitalization, transplantation and worsening of clinical outcomes.

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Conversations in Drug Development Trends

OCTOBER 14, 2024

With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world. Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential.

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The Pharma Data

JULY 3, 2025

The results, recently published in the International Journal of Molecular Sciences , come from a collaborative effort between researchers at Erlangen University Hospital and Berlin-based biotech firm AudioCure. The potential of AC102 to treat both hearing loss and tinnitus sets it apart from other investigational therapies.

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Drug Target Review

MAY 23, 2025

In the ever-evolving landscape of immuno-oncology, managing the side effects of advanced therapies has become just as important as enhancing their efficacy. Existing therapies, such as tocilizumab, work by blocking specific inflammatory pathways but often come with limitations.

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Drug Target Review

FEBRUARY 1, 2024

This can present challenges when attempting to recruit an enriched patient population for clinical trials. Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? The lack of heterogeneity can introduce significant risk into mid- to late-stage studies.

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Drug Target Review

DECEMBER 16, 2024

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.

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PPD

MAY 18, 2023

Yet, four out of five care partners of people with neurodegenerative disorders have never been informed of clinical trial opportunities , according to a survey of more than 250 caregivers conducted by PPD, the clinical research business of Thermo Fisher Scientific. There’s little time to lose.

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The Pharma Data

JUNE 9, 2025

This new monoclonal antibody therapy is designed to prevent lower respiratory tract disease caused by respiratory syncytial virus (RSV) in neonates and infants born during or entering their first RSV season. RSV disease is the leading cause of infant hospitalization in the U.S. Key results from the CLEVER trial include: A 60.5%

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Conversations in Drug Development Trends

JULY 3, 2025

Approximately two in 10 patients with hematologic malignancies are estimated to be eligible and able to receive cell therapy. The REMS Burden Explained Once deemed necessary for managing the risks associated with CAR T therapies, the REMS requirements imposed significant operational burdens on treatment centers.

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Broad Institute

NOVEMBER 25, 2024

As a proof of concept, a drug designed to target a newly discovered biological node is showing efficacy in treating rare genetic diseases in the kidney, the eye, and the brain and is now making its way to clinical trials in collaboration with a pharmaceutical partner. You can learn more about how clinical trials work here.

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Drug Target Review

DECEMBER 16, 2024

This field allowed me the privilege of tending to children in the hospital and building long-term relationships with them and their families. In addition to this, I also chaired national trials evaluating an anti-GD2 antibody in pediatric osteosarcoma.

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Drug Target Review

JUNE 19, 2025

Bazan sees it as part of a broader shift towards combination therapies – an approach that will likely define the next generation of pain management. South Rampart Pharma has completed its Phase 1 clinical trial, demonstrating strong safety, tolerability and pharmacokinetics. From bench to bedside: what is next? Dr Bazan holds a B.S.

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Clinical Trials Molecular Biology Treatment Trials 52

Conversations in Drug Development Trends

OCTOBER 14, 2024

With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world. Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential.

Research 59

Research Clinical Trials Therapies Immune Response 59

The Pharma Data

DECEMBER 28, 2020

LY-CoV555 Not Efficacious for Hospitalized Patients With COVID-19. 28, 2020 — The neutralizing monoclonal antibody LY-CoV555, given in conjunction with remdesivir, is not efficacious for hospitalized patients with COVID-19 without end-organ failure, according to a study published online Dec. Professional. MONDAY, Dec.

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The Pharma Data

JUNE 11, 2025

Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.

Disease 40

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Broad Institute

AUGUST 19, 2024

Jude Children’s Research Hospital have discovered that oleic acid, one of the most abundant fatty acids in the body, restores a healthy balance of vaginal microbes in a laboratory model of BV. At the Ragon Institute, Kwon’s lab is working to move this research toward a human clinical trial. “We Paper cited Zhu M et al.

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The Pharma Data

NOVEMBER 30, 2020

The UK-based RECOVERY trial, which is evaluating multiple treatments for COVID-19, will add the inexpensive anti-inflammatory drug colchicine to the list of therapies being tested in hospitalized patients. They said it is likely to be several months before colchicine’s efficacy is known. Source link.

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KIF1A

APRIL 25, 2024

She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane. Sloane is an adorable 3 year old with KAND. Hello KIF1A Community! We are also paying for our housing.

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Drug Target Review

JANUARY 12, 2024

This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Therefore, having biomarker information is useful for separating individuals and ensuring that you treat an individual as an individual and can tailor their therapy. Most drugs do not work in all people.

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SCIENMAG: Medicine & Health

JUNE 14, 2023

Three young patients with relapsed T-cell leukaemia have now been treated with base-edited T-cells, as part of a ‘bench-to-bedside’ collaboration between UCL and Great Ormond Street Hospital for Children (GOSH).

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Olympian Clinical Research

OCTOBER 14, 2021

If you’re thinking about joining a clinical trial, there are many benefits you could look forward to. But how can you tell if clinical trials are right for you? We’ve outlined five reasons why joining a clinical trial might be in your best interest. You’re Helping to Advance Medical Knowledge.

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Advarra

MARCH 6, 2025

For more than 35 years, Advarra has been committed to protecting the rights and welfare of clinical trial participants while helping to improve healthcare outcomes, advancing medical knowledge, and bringing innovative, life-extending treatments to market that benefit millions of patients worldwide.

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The Pharma Data

JUNE 11, 2025

The data come from Phase 2 trials evaluating its investigational therapy VYVGART® (efgartigimod) for two hard-to-treat autoimmune conditions: idiopathic inflammatory myopathies (IIM or myositis) and Sjogren’s disease (SjD). Luc Truyen, Chief Medical Officer at argenx.

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The Pharma Data

JANUARY 17, 2021

The Phase IIa POC studies will be conducted after a Phase Ib dose escalation study evaluating the tolerability and activity of proenzyme therapy in patients with advanced solid tumors is completed, planned for 2021, at the Peter Mac Cancer Center, Melbourne, Australia. About Propanc Biopharma, Inc. Propanc Biopharma, Inc.

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The Pharma Data

DECEMBER 17, 2020

(NASDAQ: REGN ) today announced that the New England Journal of Medicine (NEJM) has published initial clinical data from an ongoing seamless Phase 1/2/3 trial of the antibody cocktail casirivimab and imdevimab in non-hospitalized patients with COVID-19. “The investigational cocktail is now available to indicated high-risk U.S.

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The Pharma Data

DECEMBER 3, 2020

The approval was based on data from the Phase 3 APeX-2 trial, which showed that ORLADEYO could significantly reduce attacks at the 24-week mark. Subjects who completed 48 weeks of treatment in the Phase 3 trial saw reductions in their HAE attack rates, from an average of 2.9 The reduction was sustained through 48 weeks in the study.

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Advarra

FEBRUARY 4, 2025

At a busy research site, a clinical trial is ready to launch but its stuck in limbo over a single paragraph in the informed consent form (ICF). While the trial waits for approval, patients who are eager for a new treatment face delay. Language relating to the potential cost of participation. Age isnt the only factor to consider.

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The Pharma Data

AUGUST 7, 2020

As the pharma industry stands firm in its commitment to advance the sector to fight Covid-19, news has emerged from the European Commission who intend to streamline the development of therapies using genetically modified organisms to treat Covid-19. Regenerative medicine and advanced therapies thriving despite Covid-19 disruption.

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The Pharma Data

APRIL 19, 2022

Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

FDA Approval 52

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The Pharma Data

JUNE 27, 2025

This data includes results from two pivotal studies: Part C of the ongoing DEVOTE clinical trial and the final eight-year data from the NURTURE study. Jude Children’s Research Hospital. The findings were unveiled during the annual SMA Research & Clinical Care Meeting, hosted by Cure SMA in Anaheim, California.

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Perficient: Drug Development

JANUARY 14, 2025

With a background in marketing at a rural community hospital, Kristen has a deep understanding of the patient experience and the business of providing great care. These features will contribute to clinical, medical, and commercial ROI by accelerating products to market and getting patients the therapies they need faster.

Science 52

Science Compliance Clinical Trials Therapies 52

The Pharma Data

APRIL 3, 2021

The CoVIg-19 Plasma Alliance today announced that the Phase 3 Inpatient Treatment with Anti-Coronavirus Immunoglobulin (ITAC) clinical trial sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), did not meet its endpoints. About the ITAC Trial.

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Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes. Adapted from a press release issued by Brigham and Women's Hospital.

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The Pharma Data

JUNE 12, 2025

The positive results from APPULSE-PNH further reinforce Fabhalta’s ability to provide clinically meaningful improvements in Hemoglobin in PNH patients who previously may not have adequately responded to standard C5 inhibitor therapy — or who may wish to discontinue infusion therapy in favor of a convenient, oral option.”

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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Cytel

FEBRUARY 20, 2024

Negative binomial probability distribution can be used to model the number of times a particular outcome occurs during a clinical trial. Here, I explain this statistical methodology and its application in adaptive group sequential clinical trial designs.

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The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

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