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CRISPR-based gene editing shows promise in treating rare form of blindness

About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a paper published in the New England Journal of Medicine.

6 May 2024

MedStar Washington Hospital Center enrolls first U.S. patient in gene-editing therapy trial for heart disease

The first U.S. heart patient involved in a study for a new gene-editing therapy directly infused inside the body has successfully been enrolled at MedStar Washington Hospital Center.

6 May 2024

Plant-based medication may be an effective therapy to help people quit vaping

Eleven million U.S. adults use e-cigarettes to vape nicotine, and about half of them say that they want to stop, but many have trouble doing so because nicotine is an addictive drug.

6 May 2024

Measles and rubella vaccine delivered via microarray patch shows promising results

The phase 1/2 randomized trial compared results from the measles and rubella vaccine delivered by a microarray patch, a small sticking plaster-like device with an array of microscopic projections that painlessly penetrate the skin and deliver the vaccine, or by conventional injection with a needle and syringe.

29 Apr 2024

Majority of AI clinical trials report positive outcomes, yet concerns over generalizability persist

The state of randomized controlled trials (RCTs) for artificial intelligence (AI) algorithms in clinical practice.

29 Apr 2024

Single dose of malaria monoclonal antibody 77% effective in children, trial in Mali shows

One injected dose of an experimental malaria monoclonal antibody was 77% effective against malaria disease in children in Mali during the country's six-month malaria season, according to the results of a mid-stage clinical trial.

26 Apr 2024

First UK real-world study shows promise for sacituzumab govitecan in metastatic breast cancer

The real-world safety and efficacy of sacituzumab govitecan (SG) against metastatic triple-negative breast cancer.

26 Apr 2024

CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patients

In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

24 Apr 2024

Novel immunotherapy shows promise for high-risk sarcomas: HEROS 2.0 trial results published

Researchers at Texas Children’s Cancer Center and the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist published results of a phase I clinical trial of a novel immunotherapy for high-risk sarcomas in the journal Nature Cancer.

24 Apr 2024

WOO Network trials investigate innovative treatments for liver, head and neck cancers

A new round of clinical trials supported by the Ontario Institute for Cancer Research (OICR) will harness a unique opportunity to test some of the newest treatment strategies for a range of different cancers.

18 Apr 2024

Phase 2 trial reveals Lixisenatide may reduce motor disability in Parkinson’s patients

A trial of lixisenatide to assess its effects in early Parkinson’s disease.

18 Apr 2024

Phase 3 trial shows overall survival benefit from adjuvant therapy in kidney cancer patients

For the first time in fifty years, results from a phase 3 randomized, placebo-controlled trial have shown an overall survival benefit from an adjuvant therapy in patients with kidney cancer.

17 Apr 2024

Vosoritide's first phase 2 study shows increased growth in children with hypochondroplasia

Vosoritide's first global phase 2 study showed an average increased growth rate of 1.8 cm per year in children with hypochondroplasia, a genetic cause of short stature in children, according to researchers from Children's National Hospital.

13 Apr 2024

Home-administered nasal spray could be safely used to treat recurrent rapid heart rhythms

A clinical trial led by Weill Cornell Medicine investigators showed that a nasal spray that patients administer at home, without a physician, successfully and safely treated recurrent episodes of a condition that causes rapid abnormal heart rhythms.

9 Apr 2024

Personalized anti-tumor vaccine enhances immunotherapy for liver cancer

Adding a personalized anti-tumor vaccine to standard immunotherapy is safe and about twice as likely to shrink cancer as standard immunotherapy alone for patients with hepatocellular carcinoma, the most common type of liver cancer, according to a clinical trial led by researchers at the Johns Hopkins Kimmel Cancer Center and its Convergence Institute.

8 Apr 2024

Ninerafaxstat shows good tolerability and safety in nonobstructive HCM patients

The investigational drug ninerafaxstat showed a good tolerability and safety profile, along with evidence of improvements in symptoms and exercise capacity among people with nonobstructive hypertrophic cardiomyopathy (HCM), according to results of a 12-week phase 2 trial presented at the American College of Cardiology's Annual Scientific Session.

8 Apr 2024

Treating early-stage heart failure in diabetic patients: Insights from ARISE-HF trial

One of the first studies to attempt to treat early-stage heart failure in patients with Type 2 diabetes did not meet its primary endpoint, but a preplanned subgroup analysis found a statistically significant effect of treatment in patients who were not taking SGLT2 inhibitors or GLP-1 RAs, which are antidiabetic medications that also have benefits for the heart.

8 Apr 2024

Investigational drug plozasiran shows promise in reducing severe hypertriglyceridemia

In patients with severely elevated triglyceride levels at risk for developing acute pancreatitis, the investigational drug plozasiran reduced triglyceride levels by an average of 74% after 24 weeks of use without causing any significant safety concerns, according to research presented at the American College of Cardiology's Annual Scientific Session.

8 Apr 2024

Novel combination therapy shows promise in difficult-to-treat endometrial cancer, study finds

In a small, investigator-initiated phase 2 study by Dana-Farber Cancer Institute investigators, a novel combination of an antibody-drug conjugate and an immune checkpoint inhibitor showed notable activity in pre-treated patients with a difficult-to-treat form of endometrial cancer.

5 Apr 2024

Clinical trial to test the feasibility of using at-home cancer immunotherapy

A clinical trial from Keck Medicine of USC will test the feasibility of treating non-small cell lung cancer with immunotherapy provided at home.

28 Mar 2024

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Drug Trial News

CRISPR-based gene editing shows promise in treating rare form of blindness

MedStar Washington Hospital Center enrolls first U.S. patient in gene-editing therapy trial for heart disease

Plant-based medication may be an effective therapy to help people quit vaping

Measles and rubella vaccine delivered via microarray patch shows promising results

Majority of AI clinical trials report positive outcomes, yet concerns over generalizability persist

Single dose of malaria monoclonal antibody 77% effective in children, trial in Mali shows

First UK real-world study shows promise for sacituzumab govitecan in metastatic breast cancer

CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patients

Novel immunotherapy shows promise for high-risk sarcomas: HEROS 2.0 trial results published

WOO Network trials investigate innovative treatments for liver, head and neck cancers

Phase 2 trial reveals Lixisenatide may reduce motor disability in Parkinson’s patients

Phase 3 trial shows overall survival benefit from adjuvant therapy in kidney cancer patients

Vosoritide's first phase 2 study shows increased growth in children with hypochondroplasia

Home-administered nasal spray could be safely used to treat recurrent rapid heart rhythms

Personalized anti-tumor vaccine enhances immunotherapy for liver cancer

Ninerafaxstat shows good tolerability and safety in nonobstructive HCM patients

Treating early-stage heart failure in diabetic patients: Insights from ARISE-HF trial

Investigational drug plozasiran shows promise in reducing severe hypertriglyceridemia

Novel combination therapy shows promise in difficult-to-treat endometrial cancer, study finds

Clinical trial to test the feasibility of using at-home cancer immunotherapy

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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