Drug Target Review

JUNE 5, 2025

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease 52

Disease Treatment Therapies Nurses 52

Antidote

SEPTEMBER 16, 2024

Receiving a Parkinson’s diagnosis is a life-altering event that nearly 1 million people in the United States have experienced. Parkinson’s disease symptoms can vary from person to person. Receiving a diagnosis often requires several steps.

Disease 114

Disease Research 114

Broad Institute

FEBRUARY 25, 2025

Rare genetic diseases are collectively common: they affect 1 in 10 people in North America. While more than 8,000 genes are known to drive these diseases, fewer than 500 have an available treatment. For more information visit: Rare Disease Day

Disease 52

Disease Treatment 52

Drugs.com

JANUARY 31, 2024

31, 2024 -- A disturbing number of people sick with an infectious disease conceal their illness to avoid missing work, travel or social events, new research reveals.About three in four people (75%) had either hidden an infectious. WEDNESDAY, Jan.

Disease 189

Disease Research 189

Drug Channels

APRIL 4, 2022

Rare Disease Innovation & Partnering Summit. Hybrid Event May 17-19, 2022 | Boston, MA www.informaconnect.com/rare. Don’t miss the Rare Disease Innovation & Partnering Summit, coming up May 17-19, 2022 in Boston, MA. Visit www.informaconnect.com/rare for further details and to register. d/b/a Drug Channels Institute.

Disease 98

Disease Therapies FDA Trials 98

Science Daily: Pharmacology News

AUGUST 15, 2023

The study estimated a reduction in cardiovascular disease risk of 1.8% (from 10.15% to 8.34%), projecting up to 1.5 million cardiovascular events could be potentially prevented in 10 years. A popular weight loss medication may prevent up to 1.5

Disease 188

Disease 188

Chemical Biology and Drug Design

MAY 11, 2023

The figure depicts the neuroprotective role of crocin in Parkinson's disease. Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects.

Disease 100

Disease Research Clinical Trials Treatment 100

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.

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Clinical Development Clinical Trials Clinical Pharmacology Trials 69

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.

Disease 105

Disease Hospitals Medical Schools Therapies 105

SCIENMAG: Medicine & Health

JANUARY 12, 2024

12, 2024) — The University of Kentucky will mark a decade of raising awareness and continuing its effort to warn women about the dangers of heart disease with the annual Healthy Hearts for Women Symposium. LEXINGTON, Ky. Credit: Peera_Sathawirawong, iStock/Getty Images Plus LEXINGTON, Ky.

Disease 82

Disease 82

Fierce BioTech

SEPTEMBER 20, 2023

In this podcast interview, Dan Fisher, managing director and practice lead for IPM.ai, explains the use of artificial intelligence for stitching together key medical events that may happen over a r | IPM.ai helps brands assess a unique patient population, accelerate clinical trial recruitment, and optimize commercial outcomes

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Clinical Trials Disease Trials 105

Chemical Biology and Drug Design

FEBRUARY 13, 2024

Alzheimer's disease (AD) is a common age-related neurodegenerative brain disorder characterized by the impairment in memory and other cognitive functions. Abstract Alzheimer's disease (AD) is a common age-related neurodegenerative brain disorder characterized by the impairment in memory and other cognitive functions.

Disease 100

Disease Treatment Therapies Research 100

Drug Discovery Today

JULY 29, 2022

Geneva-based event pledges five Swiss francs per visitor to support new drug development project

Disease 113

Disease Research Drug Development Drugs 113

FDA Law Blog: Biosimilars

MARCH 7, 2024

recognition of Rare Disease Day. NORD’s announcement followed in the footsteps of European rare disease patient organization, Eurodis , who had celebrated the first Rare Disease Day the year before. Time flies when your goal is to support the thirty million people that suffer from rare diseases.

Disease 69

Disease Government FDA Drugs 69

Drugs.com

APRIL 15, 2024

MONDAY, April 15, 2024 -- Lucid episodes are an unexpected occurrence among people with late-stage Alzheimer’s disease and related dementias.But these spontaneous events -- in which a person temporarily regains an ability to communicate that.

Research 130

Research Disease 130

FDA Law Blog: Drug Discovery

JANUARY 30, 2025

This target completion date is informed by the natural history of the disease, availability of alternative treatment, anticipated recruitment timeline, and the projected timeline for efficacy analysis(es); 2) the sponsors progress and plans for postapproval conduct of the trial provide sufficient assurance to expect timely completion of the trial.

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FDA Trials Disease Drugs 105

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.

Clinical Trials 52

Clinical Trials Disease Trials Therapies 52

KIF1A

APRIL 13, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. In cases of hypoxic-ischemic encephalopathy (HIE), the developing child’s brain is damaged by an event of restricted bloodflow and loss of oxygen.

Disease 111

Disease Research Science 111

Elrig

MARCH 25, 2025

Prof Rory Johnson, Associate Professor, University College Dublin, and Dr Shalini Andersson, Vice President Nucleic Acid Therapeutics, AstraZeneca will lead this years event focussed on drugging the undruggable.

RNA 59

RNA Bioinformatics Science Pharmacokinetics 59

Advarra

NOVEMBER 14, 2024

The DSMB’s primary responsibilities include : Monitoring patient safety: Ensuring participants are not exposed to undue risk and adverse events are promptly identified and addressed. This may include differences in standard of care, acceptable practices, and the like.

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Clinical Trials Trials International Clinical Research 97

Broad Institute

MAY 21, 2025

The score, called an MM-like score, assesses the severity of disease and risk of progression to active cancer, with higher scores indicating faster progression. The MM-like score defined in this study focuses on the presence and progressive evolution of genetic abnormalities to assess the state of the disease.

DNA 52

DNA Disease Bioinformatics Hospitals 52

Drug Target Review

APRIL 11, 2024

Targeting pathways involved in SMC phenotypic modulation holds promise for developing novel therapeutic approaches for cardiovascular diseases. Can you explain the methodology used to investigate the genetic variants regulating extracellular matrix (ECM) secretion in SMCs, and predict proteins associated with vascular disease?

Disease 59

Disease Engineering Regulations Clinical Research 59

Advarra

FEBRUARY 22, 2024

Assessing and reporting adverse events (AEs) in clinical trials is critical to ensuring the study is as safe as possible and the participants have the most up to date information so they can decide whether to continue their participation in the research study. These responsibilities and reporting requirements are outlined in U.S. regulations.

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Clinical Trials Drugs FDA Research 52

Drug Target Review

SEPTEMBER 9, 2024

functions to reset the immune system and potentially provide remission for allergic diseases? In allergic diseases, APCs identify and present unknown antigens to the immune system. This can lead to inadequate long-term effectiveness with low rates of disease remission. They also require frequent administration.

Disease 64

Disease Immune Response Clinical Trials Treatment 64

Conversations in Drug Development Trends

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

Clinical Trials 52

Clinical Trials Trials Disease Drug Development 52

FDA Law Blog: Biosimilars

APRIL 13, 2025

no comparator arm), FOENIX-CCA2 was not capable of establishing improvement on time-to-event efficacy endpoints such as PFS or OS and, without an appropriate comparator, it is not possible to determine if the observed effect is attributable to LYTGOBI or to other factor(s), such as the natural history of the disease.

Drugs 52

Drugs FDA Trials Treatment 52

Drug Target Review

MARCH 17, 2025

By adjusting the activity of this receptor, these drugs can influence brain functions associated with mood, cognition and pain, positioning them as potential treatments for conditions like depression, cognitive disorders and neurological diseases. “We call this phenomenon ‘event-driven pharmacology.”

Treatment 52

Treatment Licensing Licensing Science 52

KIF1A

JULY 15, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. When the brain’s electrical activity changes enough to cause noticeable changes in behavior, we call that event a seizure. From Gschwind et al. 2023, Neuron.

Disease 98

Disease Treatment Research Therapies 98

Translation

JUNE 11, 2025

Their approach to kidney disease demonstrates both scientific excellence and a strong commitment to addressing unmet medical needs. Rege Nephro is using iPSC-derived human disease models to develop safer, more effective therapeutics that could reduce patients’ dependence on dialysis and transform long-term outcomes. expansion. “We

Science 52

Science International Disease Laboratories 52

ASPET

NOVEMBER 30, 2023

In this study, we evaluated the long-term efficacy of novel synthetic neurosteroids in preventing the development of chronic epilepsy and hyperexcitable ictal events in a rat OP model of SE. It significantly reduced the occurrence of epileptic biomarkers of HFOs and interictal spikes, indicating potential disease-modifying activity.

Disease 100

Disease Therapies Treatment 100

Drug Target Review

AUGUST 6, 2024

A number of areas represent significant opportunities for Antibody Drug Conjugates (ADCs) beyond oncology, leveraging their ability to deliver therapeutic agents specifically to diseased cells or tissues while minimising off-target effects. Oncology also offers high returns and the opportunity to address severe, life-threatening conditions.

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Therapies Pharmaceutical Companies Disease Treatment 119

Drug Target Review

JANUARY 2, 2024

We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun. Scrutinising these distinctions aids scientists in comprehending cellular operations, thereby fostering enhanced therapeutic approaches for diverse diseases.

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RNA Immune Response Therapies Disease 128

The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases. Reference: [1] Hansen, et al.

Disease 52

Disease FDA FDA Approval Treatment 52

Conversations in Drug Development Trends

APRIL 5, 2024

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

Disease 52

Disease Clinical Trials Trials Treatment 52

Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Uplifting Athletes As a 501(c)(3) nonprofit, UA harnesses the power of sport and partnerships with patient advocacy groups to fund the researchers developing datasets for rare diseases that have never been studied or understood before.

Clinical Trials 52

Clinical Trials Disease Trials Research 52

SCIENMAG: Medicine & Health

FEBRUARY 21, 2024

Using intravascular imaging to guide stent implantation during percutaneous coronary intervention (PCI) in heart disease patients significantly improves survival and reduces adverse cardiovascular events compared to angiography-guided PCI alone, the most commonly used method.

Disease 83

Disease 83

The Pharma Data

JUNE 6, 2025

Proteinuria reduction is a well-established surrogate marker for the progression of kidney disease, and this level of decline is considered both statistically significant and clinically meaningful in slowing disease progression toward end-stage kidney disease (ESKD). Treatment-emergent adverse events (TEAEs) occurred in 76.3%

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Trials Treatment Therapies FDA 40