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2020 Disease Treatment 
Drugs.com
JULY 6, 2023
Centers for Disease Control and Prevention estimates that there were nearly 68,000 new cases of acute hepatitis C in 2020 and over 107,000 newly reported cases of chronic hepatitis C? THURSDAY, July 6, 2023 -- Did you know the U.S. To help you.
299 
Drug Target Review
OCTOBER 30, 2024
Imagine being able to create an in vitro replica of a diseased organ to study the molecular mechanism underlying the illness. Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first.
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Broad Institute
APRIL 5, 2025
The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.
139 
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.
DrugBank
JULY 20, 2023
of the global population) suffer from rare conditions without effective treatments or cures. Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. At any given time, millions of people ( 3.5–5.9%
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SCIENMAG: Medicine & Health
JULY 5, 2023
New drugs are often used not only for one disease (first approved indication) but also for other diseases (supplemental indications).
67 
Eye on FDA
MARCH 7, 2024
Back in December 2023, FDA announced intention in the Federal Register and in a press release to form a new FDA Advisory Committee to be called the Genetic Metabolic Diseases Advisory Committee (GeMDAC).
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Broad Institute
JUNE 18, 2024
By Allessandra DiCorato June 18, 2024 Credit: Jon Arizti-Sanz SHINE, a rapid diagnostic test developed by Pardis Sabeti's lab in 2020, uses paper strips and CRISPR enzymes to identify specific sequences of viral RNA in samples. Tags: Infectious Disease Diagnostics Pardis Sabeti Paper cited Zhang YB, Arizti-Sanz J, et al. 2024.04.004.
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The Pharma Data
NOVEMBER 26, 2020
27, 2020 /PRNewswire/ — Protalix Biotherapeutics, Inc. Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. About Chiesi Global Rare Diseases.
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Drug Target Review
MAY 7, 2024
Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?
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National Institute on Drug Abuse: Nora's Blog
MARCH 6, 2025
Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&
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Drug Target Review
OCTOBER 5, 2023
This innovative approach involves the extraction of a patient’s own stem cells from either their bone marrow or blood , followed by a course of chemotherapy and antibody treatment. The research team identified 231 individuals diagnosed with relapsing-remitting MS, with 174 of them having undergone aHSCT treatment before the year 2020.
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The Pharma Data
DECEMBER 29, 2020
30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. an international research-focused healthcare Group (Chiesi Group), today announced final study results from the BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease.
Drug Target Review
SEPTEMBER 21, 2023
The findings supported the possibility put forward by Cotton et al (2016) concerning the chance of repurposing these drugs for adulticidal treatment. The available treatment for that is minor surgery -to remove the nodules. Need for adulticidal drugs More than 99 percent of countries worldwide has been affected by this disease.
105 
The Pharma Data
APRIL 7, 2022
It may also limit coverage for any future approved treatment in the class. These coverage restrictions, including the distinction between accelerated approval and traditional approval, have never been applied to FDA-approved medicines for other disease areas. ADUHELM is indicated for the treatment of Alzheimer’s disease.
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Drug Target Review
OCTOBER 25, 2024
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.
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The Pharma Data
JANUARY 12, 2021
(NASDAQ: NARI) (“Inari”) a commercial-stage medical device company focused on developing products to treat and transform the lives of patients suffering from venous diseases, today reported preliminary unaudited fourth quarter 2020 revenue. million to $48.9 million to $48.9 About Inari Medical, Inc. Inari Medical, Inc.
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The Pharma Data
NOVEMBER 30, 2020
30, 2020 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. NASDAQ: REGN) will host a webcast on Monday, December 7, 2020 at 4:30pm EST. Management will discuss data presented at the American Society of Hematology (ASH) 2020 Annual Meeting as well as provide updates on the Company’s broader oncology and hematology portfolio.
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The Pharma Data
DECEMBER 30, 2020
The Feverish Pricing of MS Treatments. Towards the end of March, the FDA approved two new treatments for multiple sclerosis (MS): Mayzent (siponimod) and Mavenclad (cladribine). Drug treatments for MS have been climbing an expensive staircase for a number of years. Posted: December 2020. Consumer News. Source link.
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The Pharma Data
NOVEMBER 25, 2020
AHA: Ablation Better Than Meds for Initial A-Fib Treatment. 25, 2020 — Cryoballoon ablation is superior to drug therapy as an initial treatment for the prevention of atrial arrhythmia recurrence in patients with paroxysmal atrial fibrillation , according to a study published online Nov. © 2020 HealthDay. Wazni, M.D.,
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The Pharma Data
DECEMBER 17, 2020
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.
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The Pharma Data
DECEMBER 28, 2020
Triple Treatment Combo Beneficial in BRAF-Mutant CRC. 28, 2020 — Irinotecan and cetuximab combined with vemurafenib improve progression-free survival in BRAF V600E -mutated colorectal cancer (CRC), according to a study published online Dec. © 2020 HealthDay. Posted: December 2020. Professional. MONDAY, Dec.
52 
FDA Law Blog: Drug Discovery
DECEMBER 13, 2023
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.
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The Pharma Data
DECEMBER 4, 2020
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. © 2020 HealthDay. Posted: December 2020. Professional. FRIDAY, Dec.
52 
Drug Target Review
JULY 4, 2023
These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.
145 
Drug Target Review
APRIL 7, 2025
The challenge of GPCR drug discovery G protein-coupled receptors (GPCRs) are one of the most desirable and challenging target classes in drug discovery, as their mutation can lead to a wide range of diseases such as cancer, cardiovascular disorders and neurological conditions. References Sun D, et al. Acta Pharmaceutica Sinica B , 12(7), pp.
Broad Institute
OCTOBER 23, 2024
They knew that discovering the genetic cause of her disorder would help them find other people like her, help get the condition formally recognized as a new disease, and help them better advocate for research into new treatments. This is the first human disease caused by loss of one copy of a lncRNA gene.
128 
The Pharma Data
DECEMBER 15, 2020
A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.
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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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Advarra
AUGUST 16, 2022
A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.
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The Pharma Data
MAY 31, 2022
BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.
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The Pharma Data
MARCH 16, 2022
(Nasdaq: BIIB) announced that The Journal of Prevention of Alzheimer’s Disease (JPAD) today published a peer-reviewed manuscript detailing data from the pivotal Phase 3 EMERGE and ENGAGE trials for ADUHELM ® (aducanumab-avwa) 100 mg/mL injection for intravenous use in early Alzheimer’s disease.
52 
The Pharma Data
JUNE 28, 2021
Parkinson’s disease is most common neurodegenerative movement disorder / It impacts more than 10 million people worldwide/ No function-restoring therapy is currently available / Bayer is pursuing a two-pronged approach to deliver transformative therapies with one cell and one gene therapy candidate in clinical trials.
52 
Drug Target Review
SEPTEMBER 22, 2023
Moreover, their intricate 3D structure and cellular diversity offer a more precise replication of specific structures and functions, making them promising candidates in disease modelling and drug testing. Elsevier; 2020. 2020 Oct 7;21(10):571–84. 2020 Jun 8;4(9):863–74. Available from: [link] Miranda CC, Cabral JMS.
The Pharma Data
JANUARY 19, 2021
NASDAQ: REGN) today announced that it will report its fourth quarter and full year 2020 financial and operating results on Friday, February 5, 2021 , before the U.S. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases.
52 
The Pharma Data
DECEMBER 11, 2020
On 1 December 2020, the Government of Norway and WHO met virtually for their annual strategic dialogue. Key issues discussed included universal health coverage, care management and risk reduction for noncommunicable diseases (NCDs), and health emergencies preparedness and country readiness.
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The Pharma Data
NOVEMBER 27, 2020
FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. 25, 2020 (GLOBE NEWSWIRE) — Y-mAbs Therapeutics, Inc. Danyelza is administered to patients three times in a week in an outpatient setting and the treatment is repeated every four weeks. NEW YORK, Nov.
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The Pharma Data
DECEMBER 8, 2020
Blood disease trial participants given Vertex’s and CRISPR Therapeutics’ CRISPR/Cas9-based gene-editing therapy CTX001 demonstrated a “consistent and sustained response” to the treatment, according to new phase 1/2 trial data the companies have announced. To date, a total of 19 trial participants have been dosed with the gene therapy.
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The Pharma Data
JANUARY 14, 2021
Department of Health and Human Services’ (HHS) decision to effectively remove the “X-waiver” requirement for physicians, expanding their ability to utilize medication-assisted treatment (MAT) for patients struggling with opioid use disorder (OUD). Added Dr. Rosenberg, “This is a great day for our patients.
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The Pharma Data
JANUARY 5, 2021
RAD011 is a pivotal-trial ready synthetic cannabidiol oral solution with potential utilization in multiple endocrine and metabolic orphan diseases. As part of this transaction, the Company expects to take a one-time charge of up to $16 million in the fourth quarter of 2020. Disease Highlights. Theresa Strong, Ph.D.,
52 
Drug Target Review
MARCH 19, 2024
While there has been some epidemiological support for vitamin A or deficiency in observational studies, we supported this role for the vitamin in schizophrenia using genetic approaches back in 2020. In other words, a disease state may cause a change in vitamin A levels rather than the vitamin A levels causing a change in disease state.
105 
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. How does SRP-001’s safety profile, especially concerning hepatotoxicity and nephrotoxicity, compare to other common pain medications?
59 
The Pharma Data
DECEMBER 2, 2020
3, 2020 09:00 UTC. In the hamster challenge model, even low MTX-COVAB (5 mg/kg preventive; 10 mg/kg therapeutic) protected against weight loss, a key indicator of disease severity. Compared to the treatment group, control animals lost a mean of more than 15% of their body weight. “In Source link.
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The Pharma Data
FEBRUARY 4, 2021
3, 2021 /PRNewswire/ — AbbVie (NYSE: ABBV) announced financial results for the fourth quarter and full year ended December 31, 2020. AbbVie hosted an Immunology Strategic Update event on December 14, 2020 for members of the investment community. NORTH CHICAGO, Ill., Gonzalez , chairman and chief executive officer, AbbVie.
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