Broad Institute

SEPTEMBER 28, 2023

By Allessandra DiCorato September 28, 2023 Credit: Strittmatter Laboratory, Yale University Amyloid beta clusters (red) builds up among neurons (green) in a mouse model of Alzheimer’s disease. The scientists identified a suite of changes in cells unique to the early stages of Alzheimer’s, including some not seen before in animal studies.

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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.

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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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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.

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The Pharma Data

JANUARY 4, 2021

Investors can pre-register for the event here. Eduardo Schiffrin, Medical Director, will participate in a panel discussion at the LifeSci Partners 10 th Annual Healthcare Corporate Access Event on Friday, January 8 from 11am Eastern Standard Time. Investors can pre-register for the event here. LAUSANNE, Switzerland, Jan.

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Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Miraculously, there was an approved treatment. This treatment only exists because a small charity in the ’70s donated the funds needed for the researcher to discover it. Uplifting Athletes (UA) is on a mission to change that. It saved my life.”

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Broad Institute

NOVEMBER 6, 2024

For some patients, this treatment results in striking and long-lasting remission — but for a subset, it triggers complications ranging from mild rash to potentially deadly brain and heart inflammation. With immunotherapy, it awakens your immune system in a way that’s like a fire in a bush — it doesn’t stop.

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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.

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The Pharma Data

MARCH 28, 2022

Kerendia ™ (10 mg or 20 mg), a non-steroidal, selective mineralocorticoid receptor (MR) antagonist, is approved for the treatment of chronic kidney disease and type 2 diabetes, excluding patients with end-stage renal disease or on dialysis. Finerenone is different to existing CKD in T2D treatments.

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The Pharma Data

JULY 12, 2021

Finerenone 10 mg or 20 mg is indicated to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D).

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Olympian Clinical Research

SEPTEMBER 16, 2022

million Americans were living with Alzheimer’s disease in 2021, according to the Centers for Disease Control and Prevention (CDC). Alzheimer’s disease is the seventh leading cause of death in the United States and is a devastating disease that can rob people of their memories and lives. Eating a Healthy Diet.

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The Pharma Data

JUNE 29, 2021

If approved, Forxiga has the potential to change the treatment paradigm for millions of people in the EU suffering from chronic kidney disease. CKD is a serious, progressive condition defined by decreased kidney function and is often associated with an increased risk of heart disease or stroke.

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The Pharma Data

AUGUST 9, 2021

Approval based on unprecedented DAPA-CKD Phase III data is the most significant advancement in chronic kidney disease in more than 20 years. CKD is a serious, progressive condition defined by decreased kidney function and is often associated with an increased risk of heart disease or stroke.

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The Pharma Data

DECEMBER 29, 2020

(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,

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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.

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The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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Broad Institute

NOVEMBER 6, 2024

Launched in 2017, this is a first-of-its-kind program in North America focused on improving the diagnosis, treatment, and understanding of serious immunotherapy complications, which can affect nearly every organ system.

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The Pharma Data

AUGUST 30, 2021

ALXN1840 demonstrated approximately three times greater copper mobilisation from tissues than standard-of-care treatments. Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised.

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The Pharma Data

OCTOBER 27, 2020

Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). About Krabbe Disease. Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021.

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Drug Target Review

JULY 1, 2024

As a non-opioid, SRP-001 also eliminates abuse potential, positioning it as a safer and effective drug candidate for the treatment of acute and neuropathic pain and migraine headache. In Phase I clinical trials, SRP-001 showed no serious adverse events among 56 healthy volunteers, highlighting its safety. South Rampart Pharma, Inc.

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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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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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The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. Based on these data, ADT alone should no longer be considered sufficient for patients with advanced, castration-sensitive disease.”.

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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.

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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.

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The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

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DrugBank

OCTOBER 17, 2024

Bayesian adaptive randomization , for example, can dynamically allocate patients to different treatment arms based on accumulating data, ensuring that more patients receive the most promising treatment. Machine learning algorithms can be trained on historical data to identify patterns and anomalies indicating potential safety concerns.

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The Pharma Data

FEBRUARY 25, 2022

If approved, rimegepant will be the first oral CGRP receptor antagonist in the EU, and the only migraine medication approved for both acute and preventive treatment. Pfizer is proud to have a strong footprint in Europe, which will help bring this important potential new treatment option to millions of adults in Europe living with migraine.”.

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The Pharma Data

APRIL 7, 2021

Data from the pivotal Phase 3 CARDINAL study demonstrated sutimlimab inhibited C1-activated hemolysis (abnormal destruction of healthy red blood cells) within one week of treatment and had a sustained treatment effect over the course of the study. 1,2,3 CAD impacts the lives of an estimated 12,000 people in the U.S.,

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The Pharma Data

AUGUST 19, 2020

(Momenta), a company that discovers and develops novel therapies for immune-mediated diseases, in an all cash transaction for approximately $6.5 Nipocalimab recently received a rare pediatric disease designation from the U.S. Food and Drug Administration. An estimated 2.5

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The Pharma Data

AUGUST 30, 2021

Approval marks an important transformation in the treatment of more than 13 million people suffering from chronic kidney disease in Japan. CKD is a serious, progressive condition defined by decreased kidney function and is often associated with an increased risk of heart disease or stroke.

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The Pharma Data

FEBRUARY 25, 2021

Food and Drug Administration for investigational AGN-190584 (pilocarpine 1.25%) ophthalmic solution for the treatment of presbyopia. There were no treatment emergent serious adverse events observed in any AGN-190584 treated participants. The most common treatment emergent non-serious adverse events occurring at a frequency of ?5%

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National Drug & Alcohol Research Centre Blog

FEBRUARY 12, 2024

Key findings: Alcohol is the seventh leading risk factor for mortality, disease, and disability. People with clinically significant diseases may be at increased risk of alcohol poisoning. “While we know that alcohol is a leading cause of mortality, we wanted to determine who is dying from acute alcohol poisoning.

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The Pharma Data

DECEMBER 21, 2020

UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. UNITY is a biotechnology company developing therapeutics to extend healthspan by slowing, halting or reversing diseases of aging. SUZHOU, China and ROCKVILLE, Md. ,

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The Pharma Data

OCTOBER 18, 2020

ZURICH, Switzerland–( BUSINESS WIRE )– Versantis AG , a clinical-stage company developing novel therapies for orphan liver diseases, today announced that the U.S. Current treatment options are associated with poor outcomes. UCD is a rare and life-threatening condition caused by an inherited inborn error of metabolism.

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The Pharma Data

MAY 27, 2022

The FDA’s decision is informed by the results of Pfizer’s nonclinical studies and a Phase 2a clinical study of ervogastat/clesacostat, which showed that treatment with ervogastat/clesacostat reduced liver fat with a favorable safety and tolerability profile. Pfizer Inc. NYSE: PFE) today announced the U.S.

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The Pharma Data

APRIL 26, 2022

This approval follows the recent sNDA approval for Veklury for the treatment of non-hospitalized adult and adolescent patients who are at high risk of progression to severe COVID-19. For hospitalized pediatric patients who do not require invasive mechanical ventilation and/or ECMO, a 5-day treatment course is recommended.

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