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By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Its a huge privilege to have the opportunity to point these powerful new tools at our disease.
Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. Most of these conditions are genetic in origin and the majority have no effective treatment.
The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. “For diseases like Parkinson’s, it’s more than sufficient,” he explains.
By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.
Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs. By Sarah Wicks & James E.
Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.
Drug discovery is a complex and vital field that continually seeks to identify new therapeutic targets and develop effective treatments. Dewpoint Therapeutics, a platform drug discovery company founded in 2018, has been at the forefront of this exciting field. Klein notes the importance of Dewpoint’s disease-agnostic approach.
In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. million people every year.
2010 to 2018 Saw Decrease in Rate of Death for People With HIV. 19, 2020 — From 2010 to 2018, there was a 36.6 Centers for Disease Control and Prevention Morbidity and Mortality Weekly Report. percent overall during 2010 to 2018 (from 19.4 Professional. THURSDAY, Nov. 20 issue of the U.S. Bosh, Ph.D., to 8.5). .
Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . adults have been diagnosed with dry eye disease, a chronic, episodic, multifactorial disease. It also increases your risk for inflamed cornea, cornea eye disease, corneal ulcers and injuries, and eye infections. .
There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.
Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.
However, these treatments will eventually fail for the majority of patients, so there is a strong need for better CAR therapies. How might the CD5 knockout approach impact the treatment of other types of cancers beyond T-cell lymphoma?
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.
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.”
Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.
18, 2020 /PRNewswire/ — Camurus AB (NASDAQ STO: CAMX) announced today that the Swiss agency for therapeutic products, Swissmedic, has approved weekly and monthly Buvidal ® prolonged release buprenorphine for the treatment of opioid dependence in adults and adolescents from 16 years of age. LUND, Sweden , Dec. About Buvidal.
The 2018 Farm Bill. The 2018 Farm Bill removed hemp from the Controlled Substance Act. Importantly, the 2018 Farm Bill preserved FDA authority to regulate products with cannabis or cannabis-derived compounds under the Federal Food, Drug, and Cosmetic (FD&C) Act and Section 351 of the Public Health Service Act.
Alzheimer Disease Tied to Adverse Financial Events. 4, 2020 — Alzheimer disease and related dementias (ADRDs) are associated with adverse financial events years prior to clinical diagnosis, according to a study published online Nov. Professional. FRIDAY, Dec. 30 in JAMA Internal Medicine. Lauren Hersch Nicholas, Ph.D.,
Biogen’s expansion in China now includes treatment options approved for relapsing MS and spinal muscular atrophy. today announced that China’s National Medical Products Administration (NMPA) has approved TECFIDERA ® (dimethyl fumarate) for the treatment of relapsing multiple sclerosis (MS).
Alzheon licensed ALZ-801 from NeuroChem and is developing it for Alzheimer’s disease. ALZ-801 is an advanced and markedly improved candidate for the treatment of alzheimer’s disease. 2018; 32(9): 849–861. [2]. 2018 Mar;57(3):315-333. 2007 ; Kocis et al., 2016 ; Abushakra et al., Hey JA, et al. Clin Pharmacokinet.
The World Health Organization’s (WHO) Global TB Programme welcomes the results from an important study on shortened treatment for drug-susceptible tuberculosis (TB) in children, presented at the 51 st virtual Union World Conference on Lung Health. Side-effects related to treatment were few, and similar across both groups.
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.”. About ERLEADA ® (apalutamide).
million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it. The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.
The findings may have implications for our understanding of gastrointestinal diseases and endocrine disorders, as well as pancreatic development and diabetes. Further research This discovery may have implications for understanding gastrointestinal diseases and endocrine disorders. 2018 February 8 [2023 November 1];172(4):650-65.
-based subsidiary of Terumo and a global neurovascular company, announced today the FDA Approval of the PMA Supplement for the WEB 17 System, a new addition to the WEB Aneurysm Embolization System for the treatment of intracranial wide neck bifurcation aneurysms. The WEB System received its first PMA approval in late 2018.
AstraZeneca’s Biologics License Application (BLA) for tezepelumab has been accepted and granted Priority Review for the treatment of asthma from the US Food and Drug Administration (FDA). Tezepelumab received Breakthrough Therapy Designation for patients with severe asthma, without an eosinophilic phenotype in September 2018.
s (NYSE: PFE) supplemental New Drug Application (sNDA) for LORBRENA ® (lorlatinib), expanding the indication to include first-line treatment of people with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC). The FDA action also converts the 2018 accelerated approval to full approval. PRESCRIBING INFORMATION.
based clinical trials investigating interventions for high blood pressure between 2009-2018, just 52 were exclusively in Black people. While the numbers grew over that decade – from 3.9% (3 of 77 trials) in 2009 to 6.2% (9 of 146) in 2018 – the increase was not enough to be statistically significant.
25, 2020 /CNW/ – On August 21, 2020 , Health Canada approved Lynparza® (olaparib), for the treatment of adult patients with deleterious or suspected deleterious germline and/or somatic BRCA or ATM- mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with a new hormonal agent (NHA).
gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., 2018 ; Ding et al., gingivalis is associated with cardiovascular disease. Both were reversed by treatment with COR388 ( 2020 AAIC abstract ). Periodontitis has been linked epidemiologically to cognitive impairment, and P.
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.
Consequently, these drugs have limited ability to keep patients disease-free and limited impact on patient survival. Other mechanisms that cancer cells use to escape from TCE treatment are less expected. This provides ample flexibility to optimize the treatment modality to attack specific cancers. Lancet Haem 11: e693-e707.
3, 2020 (American Heart Association News) — Your chances of dying from heart disease or stroke are higher if you live in a county considered socially vulnerable due to factors such as poverty, crowded housing and poor access to transportation, new research shows. THURSDAY, Dec. More than 1 in 3 U.S.
Eli Lilly and Company blazoned that registration is now open for the CHALLENGE-MIG clinical trial, the first and only head-to- head trial comparing twoanti-calcitonin gene- related peptide (CGRP) drugs for the preventative treatment of episodic migraine in grown-ups.1 gepants) may help people achieve the pretensions that count most to them.
26, 2021 /PRNewswire/ — Dynacure , a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today that the U.S. FDA and EMA and rare pediatric disease designation by the FDA. ” The U.S.
Food and Drug Administration (FDA) and a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval of the novel combination of the cancer treatments copanlisib and rituximab. submission is for the treatment of patients with relapsed indolent B-cell non-Hodgkin’s lymphoma (B-iNHL). Fields, M.D.,
ENSPRYNG is the first and only NMOSD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training. Importantly, people with NMOSD now have the flexibility to administer treatment at home, which may alleviate the need to travel for hospital appointments.”.
Based on this recommendation, a final decision regarding approval of Tecentriq in combination with Avastin in this disease setting, along with the full details of the approved indication, is expected from the European Commission in the near future. 0.76; p<0.0001), compared with sorafenib. RECIST v1.1).
The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing eight weeks of treatment with either maribavir or investigator assigned treatment (IAT) in transplant recipients with CMV infection refractory or resistant to existing antiviral treatments (i.e.,
Today, refined versions of these human challenge studies have become standard practice in testing vaccines for vector-borne diseases (e.g., yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops.
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. ,
Credit: Allison Colorado, Broad Communications Arriving in the Bay Area after a childhood in increasingly diverse surroundings, Martin became acutely aware of health disparities and the profound differences in disease prevalence among different populations. I wanted to know how I could have any impact on studying genetic diversity.”
The Janssen Pharmaceutical Companies of Johnson & Johnson today announced preliminary data from the Phase 1 CHRYSALIS study evaluating RYBREVANT TM (amivantamab-vmjw) for the treatment of patients with non-small cell lung cancer (NSCLC) with mesenchymal-epithelial transition (MET) exon 14 skipping (METex14) mutations. 4] , [5] , [6].
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