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BioPharma Drive: Drug Pricing
MARCH 18, 2025
The case may give doctors pause before prescribing the treatment in the future, some analysts wrote. A young man treated with Elevidys died of acute liver failure.
357 
Drugs.com
SEPTEMBER 18, 2024
18, 2024 -- Therapy dogs can help boost the spirits of health care workers in the same way they brighten the moods of hospital patients, a new study shows. WEDNESDAY, Sept. The furry, four-legged friends reduced emotional exhaustion and job stress.
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Drugs.com
MARCH 20, 2025
THURSDAY, March 20, 2025 -- Doctors should prescribe triptans for migraine patients who arent receiving relief from over-the-counter (OTC) pain relievers, according to a new clinical guideline from the American College of Physicians.The r.
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BioPharma Drive: Drug Pricing
OCTOBER 10, 2024
Seven young boys given Bluebird's Skysona later developed blood cancers, findings that could shape how doctors balance the gene therapy’s risks against its benefit.
189 
BioPharma Drive: Drug Pricing
FEBRUARY 14, 2024
The company and partner Biogen say signs from payers and doctors suggest the launch of Zurzuvae, the first oral therapy for PPD, is off to a good start.
317 
BioPharma Drive: Drug Pricing
DECEMBER 13, 2023
Uptake of Casgevy and Lyfgenia may be slow despite their dramatic benefit, physicians said, citing complexities in treatment, manufacturing and reimbursement.
189 
Codon
FEBRUARY 13, 2024
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.
131 
Drug Target Review
NOVEMBER 29, 2023
Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.
102 
FDA Law Blog: Drug Discovery
DECEMBER 7, 2022
Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
119 
SCIENMAG: Medicine & Health
JULY 18, 2023
It’s time to stop prevaricating and explore the use of psilocybin—the active ingredient in ‘magic mushrooms’—and other psychedelics to ease the often overwhelming distress faced by women with late stage gynaecological cancers, urge doctors in a commentary published online in the International Journal of Gynecological Cancer.
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Drug Target Review
SEPTEMBER 1, 2023
Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.
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PLOS: DNA Science
OCTOBER 19, 2023
Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.
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FDA Law Blog: Drug Discovery
MARCH 13, 2023
To further expand patient input into product development, the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products or “OTP” (formerly the Office of Tissues and Advanced Therapies, or “OTAT”) is holding a free public workshop titled, Clinical Trials: The Patient Experience.
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SCIENMAG: Medicine & Health
JULY 28, 2023
Pearson, co-author of the paper, is now a NEWVEC post-doctoral researcher at UMass Amherst. The discovery began with an “aha” moment by then-Ph.D. candidate Patrick Pearson, who was working in Rich’s lab, along with graduate student Adam Lynch. Lynch, lead author, is now a research fellow in the Department of Veterinary and Animal Sciences.
82 
Drug Discovery Today
JANUARY 13, 2021
Mission: Cure is a coalition of patients, doctors, researchers and entrepreneurs who are committed to advancing new therapies and cures for currently untreatable diseases. Their primary disease focus is on chronic pancreatitis, a rare inflammatory condition that affects over a million people across the globe.
113 
SCIENMAG: Medicine & Health
FEBRUARY 14, 2024
For years doctors had tried to keep a patient’s recurrent drug-resistant bacterial blood infection at bay, but it kept coming back and antibiotics were no longer working. For years doctors had […] PITTSBURGH, Feb. 14, 2024 – It was a last-ditch effort. Credit: University of Pittsburgh PITTSBURGH, Feb.
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The Pharma Data
MAY 14, 2021
Product Name: Magnetic Therapy For Idiots – Natural Healing And Pain Relief With Magnetic Therapy. Magnetic Therapy For Idiots – Natural Healing And Pain Relief With Magnetic Therapy is backed with a 60 Day No Questions Asked Money Back Guarantee. Magnet Therapy: Theory and Practice. – Dr. J.
52 
PLOS: DNA Science
OCTOBER 12, 2023
In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.
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The Pharma Data
OCTOBER 19, 2020
19, 2020 — Children with autism spectrum disorder (ASD) and/or attention-deficit/hyperactivity disorder ( ADHD ) have more doctor and hospital visits during infancy, according to a study published online Oct. MONDAY, Oct. 19 in Scientific Reports. Engelhard, M.D., and ophthalmology (aOR, 3.1).
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Drug Target Review
JANUARY 12, 2024
This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Therefore, having biomarker information is useful for separating individuals and ensuring that you treat an individual as an individual and can tailor their therapy. Most drugs do not work in all people.
111 
Broad Institute
JUNE 1, 2023
The scores could help doctors identify and treat at-risk individuals earlier in pregnancy with existing but underutilized strategies such as low-dose aspirin. They also developed polygenic risk scores, which distill a person’s likelihood of developing a condition down to a single number based on their genes.
133 
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.
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Drug Target Review
SEPTEMBER 6, 2024
What trends are driving the increased use of in vivo delivery methods in gene therapy? Generally, the amount of lentiviral vector that is used for in vivo gene therapy is larger than for ex vivo use. How are new therapeutic areas emerging as potential targets for in vivo gene therapy?
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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. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane. March 2024 – The FDA approved the IND, and the administering doctor (Dr. Hello KIF1A Community!
52 
Codon
NOVEMBER 3, 2024
The experience showed me that new therapies are needed not only to meet the targets laid out by the End TB Strategy but also to prevent drug resistance from negating the effectiveness of current therapeutics. The infant experienced no adverse effects, thereby suggesting the vaccine was safe. The researchers were left with 40 drug candidates.
102 
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.
97 
Alta Sciences
JUNE 11, 2024
My Attendance at the 2024 Boston Society Cell and Gene Therapy Conference pmjackson Tue, 06/11/2024 - 20:49 , via Wikimedia Commons" data-entity-type="file" data-entity-uuid="c7a7fa8b-b2fe-4d84-a75e-d1ba3a4e2caf" src="[link] width="742" height="249" loading="lazy" /> Ecm85, CC BY-SA 3.0
52 
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.
133 
The Pharma Data
NOVEMBER 20, 2020
20, 2020 — Hormone therapy can be a lifesaver for men with prostate cancer , but it also appears to put some at increased risk of heart problems, a new study reports. ” Doctors treating prostate cancer patients with poor heart health need to be vigilant regarding heart-related side effects of ADT, said Dr. . FRIDAY, Nov.
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.
110 
The Pharma Data
DECEMBER 9, 2020
9, 2020 — Side effects of radiation therapy in breast cancer patients are often missed by doctors, U.S. Of the more than 5,500 patients who reported at least one substantial side effect during radiation therapy, more than 53% had under-recognition of at least one of the four side effects. WEDNESDAY, Dec.
52 
Drug Target Review
JANUARY 23, 2024
Cell-based therapies, such as tumour-infiltrating lymphocyte (TIL) therapy, have also drawn significant attention following promising results in a melanoma trial. About the author Dr Espen Basmo Ellingsen University of Oslo Dr Ellingsen is a Medical Doctor and holds a PhD in cancer immunology from the University of Oslo.
122 
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.
52 
Antidote
FEBRUARY 22, 2024
Behind every new treatment is the incredible team of doctors, researchers, patients, and clinical trial specialists who worked on the research that made it possible.
Advarra
MARCH 10, 2023
Considered a rare disease, research in finding better therapies – and possibly even a cure – for SCD may be competing for funding with conditions impacting broader populations. These are reasons for biotech organizations to renew a focus on improving diversity in clinical research while bringing more sickle cell gene therapies to market.
98 
Drug Target Review
FEBRUARY 29, 2024
Through further research and work as a principal investigator on clinical studies for several central nervous system indications, I laid the groundwork for a career in gene therapy drug development. AskBio) I continue to work to bring innovative gene therapies to patients in need. In my current role at Asklepios BioPharmaceutical, Inc.
111 
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. Additionally, I get to analyze study data and learn more about the specific therapy that we evaluated. Get to know Ashley further with this Q&A: What inspired you to choose this profession?
98 
Drug Target Review
DECEMBER 28, 2023
In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. However, if the therapy targets only the subclonal (branch) mutations, this will result in the mere ‘pruning’ of specific branches rather than elimination of the whole cancer.
105 
Broad Institute
DECEMBER 6, 2023
In the future, when doctors give patients cytokine therapies targeting the immune system, they could potentially look in the Immune Dictionary for expected cell-level immune responses. There are many open questions, and having this foundational reference can significantly improve our understanding of the immune system.
138 
Drug Target Review
DECEMBER 12, 2023
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. During his post-doctoral work at PSI, he introduced the novel approach using transglutaminases for antibody conjugation that led to the discovery of the Araris ADC Technology.
114 
Drug Target Review
JUNE 29, 2023
I think the cell-therapy field has evolved a lot – and is continuing to do so – in the time I’ve been working with this. Regarding the NY-ESO programme that we worked on, I started work on that in 2002 before we even knew that we were going to do cell therapy. I mean, I think cell-therapy is an great place to be.
115 
The Pharma Data
NOVEMBER 17, 2020
“The findings from Olmsted County provide evidence for broad and delayed effects of early-life antibiotic exposures, and should change doctors’ practices in how often they prescribe antibiotics, especially for mild conditions,” a coauthor said in a statement. Abstract/Full Text. © 2020 HealthDay. All rights reserved.
52 
Broad Institute
OCTOBER 11, 2023
But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. His mother had a presentation of the disease that suggested her immune system was already on the job.
137 
Codon
JULY 27, 2023
But what happens if you restore these cone cells, using gene therapy? There’s a TV show where a bunch of doctors are walking through a hospital corridor. approved a gene therapy for hemophilia A for the first time. The therapy works by replacing beta-cells with “fresh” cells taken from a, err…dead person.
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