DrugBank

JUNE 13, 2024

Similarly, for cardiovascular diseases, implantable drug-eluting stents can deliver controlled doses of anti-proliferative drugs directly to the site of arterial blockage, preventing restenosis (re-narrowing) and improving long-term integrity of the vessel. 

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Therapies Drugs DNA Treatment 98

Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? There are a lot of drug development challenges that are, in some cases, unique to neuroscience, including neurological diseases, psychiatric disorders, and rare diseases affecting the nervous system.

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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. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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Therapies Clinical Trials Immune Response Trials 103

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. Furthermore, the diagnosis of the autoimmune diseases could be a challenge and requires several kinds of evidence.

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Research Clinical Trials Therapies Immune Response 52

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

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

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. Kutlar says the possibilities of this additional therapy are exciting.

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Disease Clinical Trials Drugs Trials 80

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.

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DNA RNA Disease Hospitals 128

Broad Institute

MARCH 8, 2024

Type 2 diabetes is a disease of elevated blood sugar impacting approximately one in 10 people in the United States that can cause devastating health complications and is usually not cured. Adapted from an MGH press release. Paper cited: Smith K, Deutsch AJ, McGrail C., Multi-ancestry polygenic mechanisms of type 2 diabetes.

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Medical Schools Hospitals Disease Therapies 127

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

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. Jude Children’s Research Hospital, the Bill & Melinda Gates Foundation, and other sources. Paper cited Zhu M et al.

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Hospitals Treatment Medical Schools Science 121

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. Children and adolescents lack robust preclinical models to replicate the pathologies and provide precise and targeted therapies.

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Disease Medical Schools Pharma Companies Clinical Trials 59

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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Treatment RNA Hospitals Disease 96

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.

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

Broad Institute

OCTOBER 3, 2024

The findings point to factors involved in how neurons communicate and fire, suggesting potential targets for new therapies. Epilepsy also has several subtypes, and while one group called developmental encephalopathies have been connected to several genes, other forms of the disease are less well understood.

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DNA Doctors Treatment Disease 133

The Pharma Data

OCTOBER 20, 2020

Kidney Disease, Injury Linked to Higher Mortality With COVID-19. The researchers found that 58 percent of patients presented with COVID-19 and renal impairment (45 percent developed acute kidney injury [AKI] during their ICU stay and 13 percent had preexisting chronic kidney disease [CKD]). Professional. TUESDAY, Oct.

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Disease Therapies Hospitals Research 52

PPD

JUNE 7, 2023

Adults aren’t the only patients affected by the increasing prevalence of liver disease. The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations.

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Trials Clinical Trials Clinical Research Disease 98

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. who require oxygen therapy due to COVID-19, OR. Yancopoulos , M.D.,

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Hospitals Production Clinical Trials Virus 52

Plenge Gen

APRIL 7, 2020

Pharmacologic intervention has the opportunity to impact disease progression in the SARS-CoV-2 / COVID-19 crisis. Repurposing of approved therapies is the fastest way to impact patients today, as these medicines have regulatory approval to enable investigator-initiated trials and have a manufacturing process to ensure drug supply.

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Immune Response Disease Clinical Trials Drugs 52

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. Over the past three years in Oncology Early Development at AbbVie, I have worked on several antibody-drug conjugates from discovery to the clinic in disease areas of high unmet need.

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Medical Schools Clinical Research Drug Development Trials 52

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.

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FDA Approval Therapies FDA Hospitals 52

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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Disease Trials Treatment Clinical Trials 52

The Pharma Data

JANUARY 15, 2021

13 in the New England Journal of Medicine , involved patients with high blood pressure in the lungs caused by interstitial lung disease (ILD). It can have a range of causes, from smoking, to occupational exposure to toxins like asbestos, as well as autoimmune diseases like rheumatoid arthritis. The study, published online Jan.

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Disease Doctors Trials FDA Approval 52

PPD

AUGUST 5, 2024

Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. Composite endpoints might include factors such as hospitalization, transplantation and worsening of clinical outcomes. Historically, the available drugs and U.S. None offers a cure for PAH.

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Clinical Trials Trials Clinical Research Disease 98

The Pharma Data

DECEMBER 3, 2020

In the long-term APeX-S trial, those who completed 48 weeks of therapy had an average attack rate of 0.8 ORLADEYO is the first and only oral therapy that was designed to prevent attacks of HAE in adults and pediatric patients age 12 and older. attacks per month at baseline, to a mean of 1.0 attacks per month. attacks per month.

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FDA Approval Therapies FDA Trials 52

PLOS: DNA Science

APRIL 18, 2024

Clues to combatting a devastating disease can come from identifying people who have gene variants – mutations – that protect them, by slowing the illness or lowering the risk that it develops in the first place. from Massachusetts General Hospital, told the Alzheimer’s Association. Quiroz, Ph.D., Quiroz, Ph.D.,

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Disease Research Therapies DNA 95

Drug Target Review

OCTOBER 1, 2024

At the beginning of my career, during my scholarship, I worked for two years at a research institute where I set up a research project focused on the study of metronomic oral therapies for patients with metastatic breast cancer (BC). That paper, of which I was in the authorship, remains a current point of reference for that therapy usage.

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Doctors Therapies Science Pharma Companies 97

Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

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Small Molecule Cell Based Assays Disease Assay Development 105

The Pharma Data

MAY 5, 2022

Novartis today announced the European Commission (EC) has approved Jakavi (ruxolitinib) for the treatment of patients aged 12 years and older with acute or chronic GvHD who have inadequate response to corticosteroids or other systemic therapies.

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Treatment Disease Therapies Clinical Trials 52

The Pharma Data

JANUARY 5, 2021

RAD011 is a pivotal-trial ready synthetic cannabidiol oral solution with potential utilization in multiple endocrine and metabolic orphan diseases. We intend to use PWS as the anchor indication and will further investigate additional orphan disease opportunities in due course.”. Disease Highlights.

Disease 52

Disease FDA Approval Clinical Trials Trials 52

Broad Institute

AUGUST 16, 2023

The mitochondrial genome, which encodes just 13 proteins, is smaller and comparatively less well studied than the genome in the cell’s nucleus, even though mutations in mitochondrial DNA can cause a number of rare diseases. They found that copy number declines consistently with age, which is in line with previous studies.

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DNA Molecular Biology Disease Regulations 98

The Pharma Data

JUNE 2, 2022

Novartis today announced a partnership with the American Society of Hematology (ASH) to provide six additional African nations with technology that is already being used in Ghana to document and share the diagnosis of babies with sickle cell disease.

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Disease Medical Schools Treatment Therapies 52

The Pharma Data

SEPTEMBER 17, 2020

17, 2020 /PRNewswire/ — Eli Lilly and Company (NYSE:LLY) and Amgen (NASDAQ:AMGN) today announced a global antibody manufacturing collaboration to significantly increase the supply capacity available for Lilly ‘s potential COVID-19 therapies. INDIANAPOLIS and THOUSAND OAKS, Calif. Reese , M.D.,

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Therapies Production Government Disease 52

PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Based on our expertise and experience, we’ve developed a dynamic approach for sponsors to fortify their research into neurodegenerative diseases. Recognize and relieve the caregiver burden.

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Clinical Trials Trials Clinical Research Doctors 86

The Pharma Data

AUGUST 15, 2021

WELIREG Approved for Adult Patients With VHL Disease Who Require Therapy for Associated Renal Cell Carcinoma, Central Nervous System Hemangioblastomas, or Pancreatic Neuroendocrine Tumors, Not Requiring Immediate Surgery. inhibitor therapy approved in the U.S. VHL disease is a rare and serious condition.

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Treatment Disease Clinical Trials Therapies 52

Drug Target Review

APRIL 14, 2025

By preventing infections before they occur, these drugs reduce the need for hospital treatments, including the costly intensive care unit (ICU) care that can burden healthcare systems. Traditional biologics manufacturing can be complex and expensive, making it difficult to offer affordable therapies for widespread diseases.

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Therapies Marketing Drug Development Treatment 52

DrugBank

DECEMBER 18, 2024

These interventions target a broad spectrum of medical conditions, encompassing chronic diseases and even substance abuse. However, non-adherence to medication regimens remains a pervasive problem, leading to suboptimal outcomes and increased hospitalizations.

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Pharmaceuticals Pharmaceutical Companies Treatment Therapies 52