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.

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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.

Immune Response 52

Immune Response Therapies Treatment Clinical Trials 52

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. Only three of the CAR-NK studies were for the treatment of solid tumours. About the authors

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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. 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

Conversations in Drug Development Trends

OCTOBER 14, 2024

Fortunately, advances in clinical research are providing hope for better treatments and outcomes. Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential. One of the biggest hurdles in developing new therapies is recruiting diverse patients for clinical trials.

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Research Clinical Trials Therapies Immune Response 52

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.

Therapies 52

Therapies Clinical Trials Treatment Trials 52

DrugBank

JUNE 13, 2024

These innovative technologies are changing how medications reach their targets within the body, leading to more effective treatments with fewer side effects.   Take cancer treatment, for example. Implantable insulin pumps equipped with continuous glucose monitoring (CGM) sensors can update old diabetes treatment options.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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Therapies Treatment Engineering Molecular Biology 59

Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? All stakeholders have benefited from these innovations.

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The Pharma Data

JUNE 23, 2025

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. According to Merck, the addition of WINREVAIR to background therapy resulted in a meaningful improvement in patient outcomes, particularly in delaying disease progression and reducing the risk of severe clinical events.

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Trials Therapies Treatment Disease 40

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.

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Hospitals Treatment Medical Schools Science 121

SCIENMAG: Medicine & Health

JULY 21, 2023

The preclinical study showed that injecting ovarian tissue-derived differentiated induced pluripotent stem cells can restore hormone production and fertility in mice with premature ovarian failure related to genetic diseases and cancer treatment A new study by investigators from Brigham and Women’s Hospital, a founding members of the Mass General Brigham (..)

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Therapies Research Hospitals Treatment 80

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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Drug Target Review

JUNE 19, 2025

As the limitations of current treatments become increasingly clear, researchers are exploring new, safer approaches. His focus, however, is on addressing a critical gap in medicine: the lack of safe, non-addictive pain treatments – a challenge that has driven the development of SRP-001. “As Dr Bazan holds a B.S.

Clinical Trials 52

Clinical Trials Molecular Biology Treatment Trials 52

SCIENMAG: Medicine & Health

JULY 5, 2023

New insights explaining why some children have a longer remission than others after having cutting-edge CAR T-cell therapy for leukaemia have been revealed by researchers at UCL, Great Ormond Street Hospital, and the Wellcome Sanger Institute.

Treatment 69

Treatment Hospitals Therapies Research 69

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

Conversations in Drug Development Trends

OCTOBER 14, 2024

Fortunately, advances in clinical research are providing hope for better treatments and outcomes. Given the complexity and widespread impact of autoimmune and bone health conditions, developing new therapies is essential. One of the biggest hurdles in developing new therapies is recruiting diverse patients for clinical trials.

Research 59

Research Clinical Trials Therapies Immune Response 59

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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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

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? This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Most drugs do not work in all people.

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Doctors Clinical Trials Treatment Therapies 111

Drug Target Review

DECEMBER 16, 2024

Despite challenges as a woman in the field, Dr Hingorani built a career focused on advancing treatments for pediatric and adult cancers. This field allowed me the privilege of tending to children in the hospital and building long-term relationships with them and their families.

Medical Schools 52

Medical Schools Clinical Research Drug Development Trials 52

Drug Target Review

JULY 15, 2025

The findings underscored that automation improves data consistency and quality, thereby supporting more accurate diagnostics and personalised treatment strategies. For instance, a recent study at Heidelberg University Hospital in Germany demonstrated the patient benefits of automating NGS workflows. hours to 24 hours.

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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. Her mother, Megan, has offered to share their experience and journey with everyone to help us understand the process and to share in their hopes, fears, challenges, and successes that come along with ASO treatment. Hello KIF1A Community!

Therapies 52

Therapies FDA Approval FDA Treatment 52

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.

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DNA Doctors Treatment Disease 133

The Pharma Data

JUNE 11, 2025

The investigational therapy, an Fc-free anti-CD40L monoclonal antibody, is being developed for the treatment of systemic lupus erythematosus (SLE), a chronic autoimmune disease with limited treatment options and significant unmet need. compared to those receiving SOC alone (mean change: 5.2).

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Disease Treatment Trials Therapies 40

The Pharma Data

JUNE 12, 2025

The data show a significant and clinically meaningful improvement in patients’ hemoglobin levels after 24 weeks of treatment with Fabhalta, alongside additional health-related benefits. 2.29) after 24 weeks of treatment with Fabhalta. APPULSE-PNH demonstrated a mean improvement in Hemoglobin of 2.01 g/dL (95% CI, 1.74–2.29)

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Therapies Treatment Disease Doctors 40

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.

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Clinical Trials Disease Trials Therapies 52

The Pharma Data

NOVEMBER 23, 2020

FDA Authorizes Use of Monoclonal Antibody Treatment for COVID-19. 23, 2020 — A combination of two monoclonal antibodies was granted emergency use authorization (EUA) for the treatment of mild-to-moderate COVID-19, the U.S. Professional. MONDAY, Nov. Food and Drug Administration announced Saturday. said in a statement.

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FDA Treatment Hospitals Clinical Trials 52

The Pharma Data

OCTOBER 22, 2020

22, 2020 — Kids with autism or attention-deficit hyperactivity disorder ( ADHD ) go to the hospital more in their first year of life than children without these conditions, according to a new study. Children with ADHD had more blood transfusions, hospital admissions and emergency department visits. THURSDAY, Oct.

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Hospitals Doctors Science Therapies 52

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).

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Treatment Disease Trials Clinical Trials 40

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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Treatment Trials Hospitals Therapies 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).

RNA 40

RNA Clinical Development Science Disease 40

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.

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Disease Treatment Trials Therapies 40

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

FDA Approval Therapies FDA Hospitals 52

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

DECEMBER 27, 2020

Swedish drugmaker Vicore Pharma has reported positive results from a phase 2 study evaluating its oral therapy for treating respiratory infection in hospitalized COVID-19 patients. Source link.

Treatment 52

Treatment Hospitals Therapies Research 52

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.

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