Broad Institute

OCTOBER 3, 2024

The findings point to factors involved in how neurons communicate and fire, suggesting potential targets for new therapies. In the future, the results could also help doctors tailor treatments to a patient’s genome. Epilepsy is one of the most common neurological disorders.

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DNA Doctors Treatment Disease 133

PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

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FDA Approval Therapies FDA Disease 52

Drug Target Review

OCTOBER 9, 2023

The potential of stem cell therapies is rooted in the pluripotency of these early embryonic stem cells. Dr Ceren Pajanoja, a Doctoral Researcher at the University of Helsinki, states that this precise and efficient technique enables the identification of cell profiles directly within the original tissue.

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Therapies Doctors Disease Research 110

Drug Target Review

FEBRUARY 29, 2024

During my academic career, I had the opportunity to examine the underlying causes of neurodegenerative diseases like Parkinson’s disease and develop novel animal models to investigate potential disease-modifying therapeutics. AskBio) I continue to work to bring innovative gene therapies to patients in need.

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Therapies Disease Clinical Trials Science 111

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

Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing. We believe we are at a breakthrough moment in Alzheimer’s disease research.

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Disease Clinical Trials Therapies Science 59

Drug Target Review

DECEMBER 13, 2023

Combinatorial analytics approaches identify combinations of features that together are associated with the disease phenotype in patient sub-groups, capturing the non-linear effects of interactions between multiple genes. The combinatorial approach is considerably more sensitive than GWAS and requires much smaller patient populations.

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The Pharma Data

AUGUST 22, 2021

In the early days of the COVID-19 pandemic, doctors in Wuhan noticed something surprising. “There’s often a phenomenon that doctors report anecdotally, or that’s mentioned in passing in a particular research paper, and that provides a clue – a hook,” he said. The next challenge was to figure out why.

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Doctors Drugs Immune Response Virus 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

Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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Drug Target Review

OCTOBER 1, 2024

Since I was a child, I always wanted to be a doctor. From a human point of view, I have always considered doctors’ work as a sort of mission for the good of others. Being a doctor means giving courage and hope day in, day out to those experiencing illness. Oncology has come a long way in recent years.

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Doctors Therapies Science Pharma Companies 97

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

Alta Sciences

JUNE 12, 2024

I’ve always loved biological sciences, but I didn’t want to become a doctor and didn’t know what other careers in the field existed. Every study is different, so I am constantly learning about different diseases, therapeutic areas , and experimental methods while developing study protocols. What is one of your proudest achievements?

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The Pharma Data

DECEMBER 10, 2020

Dr. Pirozzi received a Doctorate of Medicine from Università Campus Bio-Medico di Roma in Italy, a Doctorate of Philosophy in Immunology from Sapienza Università di Roma in Italy, a Post-Doctorate degree in Immunology from the Institut Pasteur in Paris, France, and business training at the London Business School, UK.

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Disease Research Doctors Clinical Development 52

Drug Target Review

DECEMBER 12, 2023

Our linker technology platform has the potential to develop ADCs that target a wide range of different types of diseases, beyond cancer. He positioned Araris to shift the paradigm in how to think about developing targeted cancer therapies and since October 2023, he transitioned to become its CSO.

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Treatment Engineering Pharmacokinetics Doctors 114

PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. The care partners are the decision-makers for their loved ones with neurodegenerative disorders, handling everything from daily routines and medication management to doctor visits.

Clinical Trials 86

Clinical Trials Trials Clinical Research Doctors 86

The Pharma Data

NOVEMBER 17, 2020

The researchers identified associations between early antibiotic exposure and an increased risk for childhood-onset asthma, allergic rhinitis, atopic dermatitis, celiac disease, overweight, obesity, and attention-deficit/hyperactivity disorder (hazard ratios ranging from 1.20 Abstract/Full Text. © 2020 HealthDay. All rights reserved.

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Therapies Doctors Disease Research 52

Drug Target Review

SEPTEMBER 22, 2023

Organoid technologies are becoming an invaluable solution for preclinical research, with the ability to augment the development of personalised medicine, drug discovery and gene therapies. Organoids for cell therapy and drug discovery. Mice are often expensive and time-consuming to maintain and breed. Cell Stem Cell [Internet].

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Clinical Research Research Drug Development Molecular Biology 116

The Pharma Data

SEPTEMBER 23, 2020

Even with the family hardships, seeing her sister battle her illness gave Lisa the strength to face this new challenge head-on and take a leading role in her disease. I challenged my doctor quite often, always asking questions, ‘What’s next? I had to rely on the doctor for all my answers.”. What can you do for me?

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Doctors Clinical Trials Treatment Disease 52

The Pharma Data

AUGUST 31, 2020

Understanding CAR-T cell therapy in advanced B-cell blood cancers. I pressed my doctor to give me some other choice. He told me about CAR-T cell therapy. Steve , who received CAR-T cell therapy for his advanced B-cell blood cancer. Steve , who received CAR-T cell therapy for his advanced B-cell blood cancer.

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Doctors Therapies Disease Research 52

The Pharma Data

JANUARY 20, 2021

Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and granted Priority Review for Esbriet ® (pirfenidone) for the treatment of unclassifiable interstitial lung disease (UILD). In 2020, the FDA granted Orphan Drug Designation and Breakthrough Therapy Designation to Esbriet for UILD.

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FDA Disease Doctors Treatment 40

Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

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Disease Clinical Trials Trials Treatment 52

The Pharma Data

SEPTEMBER 10, 2021

More than 20 abstracts across disease states, including Parkinson’s disease, spasticity and cervical dystonia, will be presented. “Our research presented at MDS 2021 builds upon our expertise in neuroscience and reinforces our mission to advance the standards of care for people living with these debilitating diseases.”

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Doctors Clinical Trials Disease Treatment 52

KIF1A

AUGUST 20, 2024

So the pediatrician doctor requested the first cranial tomography, which also gave a normal result. Pauline is now 10 months old, undergoing physical and occupational therapy using the Cuevas method and is also having speech therapy sessions to help and strengthen the muscles involved in swallowing.

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Doctors Hospitals DNA Therapies 52

Drug Target Review

SEPTEMBER 28, 2023

I originally wanted to be a doctor but that’s a second-tier track in France. They needed reference materials for the disease in order to develop and validate diagnostic tests. NGS tools can be used to ‘read’ DNA to identify a disease subclass and detect specific mutations. I’m a CEO with a background in chemistry.

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DNA Organic Chemistry Science Virus 116

The Pharma Data

JANUARY 17, 2021

Optometrists provide comprehensive eye care, including evaluations for glasses and contact lenses and common eye diseases. They diagnose and treat eye diseases and prescribe eyeglasses and contact lenses, and they also perform eye surgery. They aren’t doctors, according to the American Association of Certified Orthoptists.

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Medical Schools Doctors Treatment Disease 52

KIF1A

JUNE 4, 2024

Hundreds of doctor appointments and uncountable exams, second and third opinions, but nothing made sense. Something that seemed so easy, became something very hard to finally do, there’s very little information about genetic disorders in Chile, so it was hard to find consensus between Elena’s doctors on which was the right test.

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Doctors Therapies Treatment Laboratories 52

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. This allowed the doctors to perform the lumbar puncture and dose administration while she was asleep and still. During sedation, Sloane also had an MRI to assess for cerebral atrophy.

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Treatment Doctors Hospitals Therapies 52

FDA Law Blog: Biosimilars

DECEMBER 4, 2023

Unusual Amounts and Dosages Dispensing high doses of opioids in quantities far exceeding the daily morphine milligram equivalent (“MME”) dose recommended by the Centers for Disease Control and Prevention (“CDC”). Complaint ¶ 57. (The Complaint ¶ 58. Providers should be aware of the other drugs prescribed to their patients. Complaint ¶ 63.

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Pharmacy Government Treatment Compliance 139

Drug Target Review

AUGUST 29, 2023

I then chose to do my doctoral research at the Perelman School of Medicine at the University of Pennsylvania, which provided me with great scientific training, critical thinking, and communication skills. In terms of my own contributions, I believe my research can in part play a role in driving future innovation in precision therapies.

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Science Disease Molecular Biology Therapies 98

KIF1A

MAY 27, 2023

Each week, Dr. Dominique Lessard and Dr. Dylan Verden of KIF1A.ORG summarize newly published KIF1A-related research and highlight progress in rare disease research and therapeutic development. And while an individual disease might be rare, collectively over 30 million people suffer from rare diseases in the United States alone.

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Science Disease Small Molecule Research 94

Drug Target Review

DECEMBER 4, 2023

Tailoring medical care to a patient’s unique genetic makeup, alongside the close analysis of disease progression, leads to more effective treatments, reduced side effects and faster diagnoses. The reason for all this attention and investment is clear: significant benefits for patients. 2023 [cited 2023 Nov 15].

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Treatment Science DNA Doctors 105

On Medicine

FEBRUARY 26, 2023

Behçet’s disease is a rare multisystem vasculitic disorder that can affect any of the blood vessels of the body; signs and symptoms are diverse among individuals and range in severity, but often present as recurrent oral aphthous ulcers, genital ulcers, or as complications involving the eyes.

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The Pharma Data

FEBRUARY 1, 2021

By getting what is known as a ‘high tibial osteotomy,’ younger patients with less severe joint damage who are physically active might be able to delay the need for a knee replacement by 10 years or more, though they may have to search for a doctor who performs the surgery. “Ask your doctor, “Would this be right for me?

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Doctors Disease Hospitals Research 52

Olympian Clinical Research

SEPTEMBER 16, 2021

Psoriasis is an autoimmune disease , which means that the body’s own immune system becomes overactive and attacks healthy tissues in the body. . Similar to a majority of autoimmune diseases, the cause is most likely genetic, but environmental triggers are required to initiate disease activity. . Causes of Psoriasis .

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The Pharma Data

AUGUST 4, 2020

It also advises doctors to go beyond simply recommending diet and exercise. Ximena Ramos-Salas, the director of research and policy at Obesity Canada and one of the guideline’s authors, said research shows many doctors discriminate against obese patients, and that can lead to worse health outcomes irrespective of their weight.

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Doctors Research Therapies Disease 52

Drug Target Review

JULY 3, 2024

With a predominantly industrial background, I attended an engineering school before embarking on a doctoral thesis in science. It was the focus of my doctoral thesis, eventually leading to the co-founding of a startup. I persevered with the support of mentors as I was determined to pursue my passion.

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