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.

Treatment 96

Treatment RNA Hospitals Disease 96

Broad Institute

JANUARY 25, 2024

They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We

Disease 124

Disease Research Science Medical Schools 124

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

Disease Treatment Trials Therapies 40

Broad Institute

FEBRUARY 7, 2024

However, researchers lack efficient ways to explore how these genetic variants are molecularly connected to cardiovascular disease, limiting efforts to develop therapeutics. The team found that a key biological mechanism involved in a rare vascular disease may influence CAD risk.

Disease 105

Disease Hospitals Medical Schools Therapies 105

Drug Discovery Today

MAY 1, 2020

Oxford BioDynamics’ EpiSwitch technology has been chosen as the biomarker platform for prognostic and predictive profiling of COVID-19 patients in the GETAFIX clinical study.Institute of Infection, Immunity and Inflammation, University of Glasgow, and NHS Scotland are leading the effort to identify patients at risk of severe immune complications and (..)

Treatment 100

Treatment Hospitals Disease 100

The Pharma Data

JUNE 12, 2025

The data, presented at the annual European Alliance of Associations for Rheumatology (EULAR 2025) congress in Barcelona, Spain, show that dapirolizumab pegol resulted in significant and clinically meaningful improvements in disease activity while addressing a major symptom that profoundly impacts patients’ daily lives — fatigue.

Disease 40

Disease Treatment Therapies Trials 40

Drug Target Review

JULY 13, 2023

Drug discovery is a complex and vital field that continually seeks to identify new therapeutic targets and develop effective treatments. Dewpoint’s breakthrough was the realisation that certain condensates are associated with human diseases. Klein notes the importance of Dewpoint’s disease-agnostic approach.

Disease 98

Disease Small Molecule Cell Biology Drugs 98

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. Importantly, the treatment also appeared to reverse the underlying physiological damage believed to contribute to tinnitus.

Clinical Trials 52

Clinical Trials Hospitals Treatment Trials 52

Broad Institute

AUGUST 19, 2024

A common fatty acid may help restore healthy vaginal bacteria after infection By Ari Navetta August 19, 2024 Breadcrumb Home A common fatty acid may help restore healthy vaginal bacteria after infection Treatments using oleic acid, a naturally occurring oil used in cells, could improve treatment of recurrent bacterial vaginosis infections.

Hospitals 121

Hospitals Treatment Medical Schools Science 121

Conversations in Drug Development Trends

OCTOBER 14, 2024

Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Fortunately, advances in clinical research are providing hope for better treatments and outcomes.

Research 52

Research Clinical Trials Therapies Immune Response 52

SCIENMAG: Medicine & Health

AUGUST 15, 2023

In a study published in the journal Genes & Diseases, researchers from Naval Medical University and Soochow University conducted an in-depth investigation into the genomic landscape of schistosomiasis-associated colorectal cancer (SA-CRC).

Research 92

Research Treatment Hospitals Disease 92

Broad Institute

OCTOBER 21, 2024

By Leah Eisenstadt October 21, 2024 Credit: Kevin Middleton, Broad Communications In the Bhattacharyya lab, researchers develop methods to quickly identify the most appropriate treatments for bacterial infections. The findings appear in the Journal of Clinical Microbiology. We absolutely couldn’t make these discoveries without them.”

RNA 115

RNA Hospitals Laboratories Medical Schools 115

Broad Institute

FEBRUARY 16, 2024

The way that clinicians subdivide diabetes patients now is based on symptoms, but in this study, the frequency of genetic risk factors seems to vary among patients with youth-onset T2D,” said Jason Flannick , Broad associate member and assistant professor at Boston Children’s Hospital and Harvard Medical School. “We

Medical Schools 133

Medical Schools Hospitals Disease DNA 133

The Pharma Data

JUNE 23, 2025

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. Participants were classified as WHO Group 1 PAH, functional class (FC) II or III, indicating moderate to severe disease at either intermediate or high risk of clinical deterioration.

Trials 40

Trials Therapies Treatment Disease 40

Drug Target Review

JULY 15, 2025

The latest technologies are empowering researchers with faster, more efficient and more accessible genomic tools, leading to richer data generation and accelerating progress in areas like cancer genomics, rare disease research and infectious disease surveillance.​ hours to 24 hours.

RNA 59

RNA DNA Laboratories Science 59

Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

Clinical Trials 52

Clinical Trials Disease Trials Therapies 52

Broad Institute

SEPTEMBER 18, 2024

By Allessandra DiCorato September 18, 2024 Credit: National Institute of Allergy and Infectious Diseases, National Institutes of Health Colorized scanning electron micrograph of Escherichia coli, grown in culture and adhered to a cover slip. Pust is in the lab of Xavier, who is co-director of its Infectious Disease and Microbiome Program.

Medical Schools 119

Medical Schools Disease Hospitals Treatment 119

Broad Institute

OCTOBER 3, 2024

Largest-ever genetic study of epilepsy finds possible therapeutic targets By Ari Navetta October 3, 2024 Breadcrumb Home Largest-ever genetic study of epilepsy finds possible therapeutic targets Scientists have uncovered new genetic links to different types of epilepsy, which could lead to more tailored treatments.

DNA 133

DNA Doctors Treatment Disease 133

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. Octavio Ramilo, Chair of the Department of Infectious Diseases at St. Key results from the CLEVER trial include: A 60.5%

FDA Approval 40

FDA Approval FDA Hospitals Clinical Trials 40

Broad Institute

OCTOBER 23, 2024

They knew that discovering the genetic cause of her disorder would help them find other people like her, help get the condition formally recognized as a new disease, and help them better advocate for research into new treatments. This is the first human disease caused by loss of one copy of a lncRNA gene.

DNA 128

DNA RNA Disease Hospitals 128

The Pharma Data

JUNE 27, 2025

Biogen Unveils New Data Underscoring the Long-Term Benefits and Advancements of Nusinersen in Spinal Muscular Atrophy Treatment Biogen , a global leader in neuroscience, has announced compelling new data that reinforces the clinical value and therapeutic potential of nusinersen, a pioneering treatment for spinal muscular atrophy (SMA).

Treatment 40

Treatment Disease Trials Clinical Trials 40

Drug Target Review

MAY 18, 2023

In sickle cell disease, however, this function is impaired, leading to a range of potential health consequences. The irregularly shaped and sticky red blood cells produced by the disease can obstruct small blood vessels, causing recurring painful attacks. Hydroxyurea, the first sickle cell drug approved by the U.S.

Disease 80

Disease Clinical Trials Drugs Trials 80

Drug Target Review

JANUARY 12, 2024

How does the integration of Real-World Data (RWD) with genomic biomarker data contribute to a more comprehensive understanding of disease progression and treatment response? Biomarker information is also useful for tracking progression of the disease and treatment response. Most drugs do not work in all people.

Doctors 111

Doctors Clinical Trials Treatment Therapies 111

KIF1A

MAY 29, 2024

Fortunately, Sloane does not currently have epilepsy, and Megan hopes this treatment will prevent it’s onset as her disease progresses. On the day of the treatment, they checked into the hospital where Sloane was administered sedation medication. The post Sloane’s 1st ASO treatment appeared first on KIF1A.

Treatment 52

Treatment Doctors Hospitals Therapies 52

The Pharma Data

JUNE 11, 2025

argenx Unveils Positive Phase 2 Results for Efgartigimod in Myositis and Sjogren’s Disease at EULAR 2025 argenx SE a global immunology company dedicated to advancing treatments for severe autoimmune diseases, presented encouraging new clinical data at the 2025 European Congress of Rheumatology (EULAR), held June 11–14 in Barcelona, Spain.

Disease 40

Disease Treatment Trials Therapies 40

The Pharma Data

JANUARY 26, 2021

Zydus Cadila Healthcare’s Desidustat has demonstrated efficacy in a phase 2b clinical trial in Mexico for COVID-19 patients hospitalized with hypoxia. Zydus has two ongoing phase 3 trials evaluating Desidustat for treatment of anemia linked to chronic kidney disease and chemotherapies. Source link.

Hospitals 52

Hospitals Clinical Trials Trials Treatment 52

The Pharma Data

JUNE 18, 2025

This leadership transition arrives at a pivotal moment in Alnylam’s evolution, as the company builds upon the recent commercial success of AMVUTTRA® (vutrisiran) —a landmark treatment for transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Garg comes at a time when the company is aggressively pursuing a broad pipeline strategy.

RNA 40

RNA Clinical Development Science Disease 40

biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Zebrafish Pioneers Advance in Pediatric Cancer Research and Treatment Increasing the survival rate is the primary goal when screening for cancer drugs.

Disease 59

Disease Medical Schools Pharma Companies Clinical Trials 59

Drug Target Review

MAY 23, 2025

As the biotech sector races to improve the tolerability of these revolutionary treatments, Poolbeg Pharma has a novel solution: an oral therapy, POLB 001, to block the development of CRS, by limiting inflammation without affecting the anti-cancer immune responses that are vital for effective immunotherapies.

Immune Response 52

Immune Response Therapies Treatment Clinical Trials 52

Broad Institute

JUNE 18, 2024

Now researchers have developed a low-cost paper strip test that could allow more patients to find out which type of flu they have and get the right treatment. They wanted to create tests that could be used in the field or in clinics rather than hospitals or diagnostic labs with expensive equipment. The Journal of Molecular Diagnostics.

Virus 144

Virus RNA Laboratories Medical Schools 144

Drug Target Review

APRIL 14, 2025

With many healthcare systems prioritising cost-effectiveness, there is limited demand for novel treatments, even though they are crucial to combatting antibiotic resistance. Traditional biologics manufacturing can be complex and expensive, making it difficult to offer affordable therapies for widespread diseases.

Therapies 52

Therapies Marketing Drug Development Treatment 52

KIF1A

JUNE 20, 2025

Wearable Digital Data Collection Is On the Horizon: Meet BioSensics KIF1A.ORG is thrilled to announce an exciting new initiative to enhance our natural history study and accelerate progress toward clinical trials: wearable digital data collection in partnership with BioSensics , a leading innovator in digital health monitoring for rare diseases.

Clinical Trials 40

Clinical Trials Trials Disease Clinical Research 40

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. Treatment outcomes for large B-cell lymphoma were dismal before CAR T-cell therapies.

Therapies 52

Therapies Clinical Trials Treatment Trials 52

Conversations in Drug Development Trends

OCTOBER 14, 2024

Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Fortunately, advances in clinical research are providing hope for better treatments and outcomes.

Research 59

Research Clinical Trials Therapies Immune Response 59

KIF1A

OCTOBER 28, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research Genetic overlap between ALS and other neurodegenerative or neuromuscular disorders How do you define a disease?

Disease 105

Disease Treatment Research Hospitals 105

The Pharma Data

OCTOBER 22, 2020

FDA Approves Veklury (remdesivir) for the Treatment of COVID-19. Food and Drug Administration (FDA) has approved the antiviral drug Veklury (remdesivir) for the treatment of patients with COVID-19 requiring hospitalization. FOSTER CITY, Calif.–(BUSINESS –(BUSINESS WIRE)–Oct.

FDA Approval 52

FDA Approval Treatment FDA Hospitals 52

The Pharma Data

JANUARY 4, 2021

Odds of Anxiety, Depression, ADHD Up With Congenital Heart Disease. 4, 2021 — Youth with congenital heart disease (CHD) have increased odds of anxiety and/or depression or attention-deficit/hyperactivity disorder (ADHD), regardless of disease severity, according to a study published online Jan. MONDAY, Jan. Gonzalez, M.D.,

Disease 52

Disease Hospitals Treatment Research 52