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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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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FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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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.

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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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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. If all goes well, her treatments will continue on a quarterly basis thereafter.

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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!

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Broad Institute

JUNE 1, 2023

Without prompt diagnosis and treatment, hypertensive disorders of pregnancy (HDPs) can lead to organ failure and potentially fatal complications. The scores could help doctors identify and treat at-risk individuals earlier in pregnancy with existing but underutilized strategies such as low-dose aspirin.

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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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Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? This growth has been influenced by the increased use of lentiviral vectors for more prevalent diseases, earlier treatment lines and broader applications. How are new therapeutic areas emerging as potential targets for in vivo gene therapy?

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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.

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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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Broad Institute

OCTOBER 11, 2023

Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. His mother had a presentation of the disease that suggested her immune system was already on the job.

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FDA Law Blog: Biosimilars

DECEMBER 4, 2023

A prescription not issued in the usual course of professional treatment or in legitimate, authorized research is not a prescription within the meaning of the CSA and the pharmacist knowingly filling it, along with the prescriber issuing it, is subject to penalties. 21 U.S.C. § 829; 21 C.F.R. Complaint ¶ 58. Complaint ¶ 59. Complaint ¶ 62.

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Olympian Clinical Research

NOVEMBER 9, 2022

Therefore, if you or a loved one is dealing with this rare condition, it’s essential to educate yourself about the condition and its potential treatments. Radiation therapy. To diagnose bullous pemphigoid, your doctor may order a blood test and take a skin biopsy. Bullous Pemphigoid Risk Factors and Causes.

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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.

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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.

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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. This cure comes with a huge caveat – only the sickest of the sick are eligible for this curative treatment. Moss, on the surface, is fairly healthy.

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The Pharma Data

DECEMBER 9, 2020

9, 2020 — Side effects of radiation therapy in breast cancer patients are often missed by doctors, U.S. It was common for treatment side effects such as pain, itchy skin, swelling and fatigue to go unrecognized. WEDNESDAY, Dec. researchers report.

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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.

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

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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. What’s more, it remains an open question as to whether our healthcare facilities are currently equipped to deliver such treatment programmes at scale.

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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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Drug Target Review

OCTOBER 24, 2023

Live Bacterial Products (LBPs) will provide patients with a desirable, credible, safe and effective treatment option. The human microbiome, a complex ecosystem of microorganisms residing within our bodies, has emerged as a promising frontier in the quest for innovative medical treatments.

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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.

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On Medicine

MARCH 22, 2023

A previous study involving brain tumor patients identified that when anti-tumor treatment is understood to be ‘the only option’, the risk and impact of side effects may not be adequately explained to and understood by patients. In some cases, photon RT can also cause abnormalities in hormone levels several years after treatment.

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The Pharma Data

SEPTEMBER 23, 2020

I challenged my doctor quite often, always asking questions, ‘What’s next? When Lisa was diagnosed, CML was still in its early days of research, and the treatments that are currently available today were not available outside of some clinical trials 2. 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

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. Stem cell-derived organoids and their application for medical research and patient treatment.

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Broad Institute

JANUARY 25, 2024

“This could potentially help with disease prevention — if we know from someone’s retinal image that they are at high risk of developing glaucoma or cardiovascular disease in the future, we could refer them for follow-up screening or preventative treatment.”

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The Pharma Data

JULY 21, 2021

A BTD is intended to expedite the development and review of medications to treat a serious medical condition and is granted when preliminary clinical evidence indicates the investigational therapy may demonstrate substantial improvement over existing therapies. PRNewswire/ — AbbVie (NYSE: ABBV) announced today that the U.S.

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The Pharma Data

JANUARY 17, 2021

The treatment could be administered in primary care centers to people who test positive for COVID-19, avoiding hospitalization due to the progression of the disease and complementing the vaccine in the early phase after vaccination. BARCELONA, Spain , Jan. 18, 2021 /PRNewswire/ — Grifols (MCE: GRF, MCE: GRF.P,

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The Pharma Data

OCTOBER 20, 2020

20, 2020 — It may seem counterintuitive, but when someone with the eating disorder anorexia nervosa is hospitalized, treatment often begins by cutting calories. Plus, with current medical technology and lab capabilities, doctors can safely monitor shifts in health and adjust treatment quickly if necessary. TUESDAY, Oct.

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The Pharma Data

DECEMBER 30, 2020

(NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced positive results from the long-term, open-label Phase 3 MOVEMENT trial of AXS-07, Axsome’s novel, oral, multi-mechanistic investigational medicine in the acute treatment of migraine.

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The Pharma Data

JANUARY 15, 2021

Food and Drug Administration (FDA) approval of Darzalex Faspro ® (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.[1]

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KIF1A

JUNE 4, 2024

“We really hope that someday there will be a treatment that can help stop the progression of the symptoms, and give Elena and us more years to enjoy together.” Hundreds of doctor appointments and uncountable exams, second and third opinions, but nothing made sense. The day we received the KAND diagnosis the world stopped.

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H1 Blog

MAY 10, 2023

Healthcare providers are often the first line of defense in ensuring that people receive the medical treatment they need. From being key opinion leaders (KOLs) for pharma treatments to providing resources outside of going to see a doctor, it’s time to recognize the role that nurses can play in contributing to better health outcomes.

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The Pharma Data

MAY 19, 2023

ABBOTT RECEIVES FDA APPROVAL FOR TACTIFLEX™ ABLATION CATHETER FOR TREATMENT OF ABNORMAL HEART RHYTHM Abbott (NYSE: ABT) today announced that the U.S. 6 “Abbott is leading the way in helping doctors manage common arrhythmias with the most holistic portfolio for this condition in the world,” said Christopher Piorkowski, M.D.,

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