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.

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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 (..)

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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.

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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

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.

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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).

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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.”

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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

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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.

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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

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.

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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.

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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

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.

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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.

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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.

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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.

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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.

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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?

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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.

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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.,

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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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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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Drug Target Review

AUGUST 29, 2024

However, these treatments will eventually fail for the majority of patients, so there is a strong need for better CAR therapies. How might the CD5 knockout approach impact the treatment of other types of cancers beyond T-cell lymphoma?

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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. GvHD occurs when donor cells see the recipient’s healthy cells as foreign and attack them.

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Drug Target Review

NOVEMBER 2, 2023

Known as mosaicism, occasionally one of those groups of cells has genetic changes that may cause disorders or diseases. Scientists from Nationwide Children’s Hospital discovered an alternate origin of brain mosaicism in some children with focal epilepsy. Epilepsy in Children: From Diagnosis to Treatment with Focus on Emergency.

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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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The Pharma Data

JANUARY 5, 2021

Centers for Disease Control and Prevention. For the study, the researchers reviewed medical records of close to 119,000 kids, aged 4 to 17, who were treated at Texas Children’s Hospital in Houston more than once between 2011 and 2016. Of those, 1,164 had congenital heart disease. TUESDAY, Jan. who reviewed the findings.

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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

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. The recommended dose of WELIREG (40 mg tablets) is 120 mg once daily until disease progression or unacceptance toxicity.

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The Pharma Data

NOVEMBER 25, 2020

The approval of remdesivir ( Veklury ) for the treatment of patients hospitalized with COVID-19 met this legal and scientific standard. This included the ACTT-1 trial sponsored by National Institute of Allergy and Infectious Disease (NIAID) and the “SIMPLE” trials (GS-US-540-5774 and GS-US-540-5773) sponsored by Gilead Sciences Inc.

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The Pharma Data

JANUARY 11, 2021

Collaboration agreement with Neurosurgery Department of Karolinska University Hospital will further enhance the usage of the company’s transformative IRRAflow system to benefit neurocritical care patients in Sweden. About Karolinska University Hospital. STOCKHOLM , Jan. STOCKHOLM , Jan. About IRRAS.

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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. Only three of the CAR-NK studies were for the treatment of solid tumours. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

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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

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.

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Medical Schools Clinical Research Drug Development Trials 52

The Pharma Data

AUGUST 22, 2021

The findings, published online in The American Journal of Geriatric Psychiatry , evaluated the long-term use of AUSTEDO with the total motor Abnormal Involuntary Movements Scale (AIMS) score, and measures of treatment success and quality of life. IMPORTANT SAFETY INFORMATION.

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The Pharma Data

DECEMBER 3, 2020

New Tau Protein is Abnormal in Very Early Alzheimer’s Disease. Investigators with the University of Gothenburg identified new forms of tau protein that become abnormal in the very early stages of Alzheimer’s disease, even before the development of cognitive problems. Kidney Disease Leading Risk Factor for COVID-related Hospitalization.

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