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Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
The data were featured as part of four oral presentations selected by the Associated Professional Sleep Societies (APSS) for their scientific innovation and clinical relevance, with Jazz accounting for all the industry-sponsored late-breaking oral presentations at the meeting.
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.
Designed using the same fundamental mechanism as Biogen’s approved SMA treatment SPINRAZA (nusinersen), salanersen represents a next-generation approach aimed at greater potency and less frequent dosing—potentially requiring only once-yearly administration. Source link
2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1]
Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). The results were remarkable for this very sick patient population, with a median OS of 14 months at the dose of 10 mg given every 2 weeks: The data supported FDAapproval, in May 2024, of tarlatamab for refractory ES-SCLC.
The study, which is the largest to date that focuses exclusively on this patient population, shows high overall response rates (ORR) alongside deep and durable responses, offering much-needed hope for individuals whose disease typically carries a poor prognosis. Head of Myeloma Unit at Tel-Aviv Sourasky Medical Center in Israel.
3] Inavolisib was approved for medical use in the United States in October 2024. [3] The difluoromethyl group can interact with the hydroxyl group presented on Ser774 (conserved) in p110, which is 3.2 3] [6] [20] [21] The drug application was granted priority review and breakthrough therapy designations by the FDA. [3]
Researchers have identified nearly 10,000 monogenic diseases , or conditions caused by errors in a single gene. Fortunately, as readily as these diseases are discovered and characterized, so too are many of their cures. Fortunately, as readily as these diseases are discovered and characterized, so too are many of their cures.
These resources provide accurate and current strategies that strongly represent FDAs standpoint and have routinely been leveraged by Premiers team of strategists when guiding sponsors through their development plans. The summary presented in the table below features some of these initiatives. Program What is it?
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. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year. tuberculosis.
2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 2] Crinecerfont was approved for medical use in the United States in December 2024. [2] Food and Drug Administration (FDA) (Press release).
FDAApproves KEYTRUDA® (Pembrolizumab) for Perioperative Treatment of Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma Merck known as MSD outside the United States and Canada, recently announced that the U.S. 0.89; p = 0.00140).
Journal of Medicinal Chemistry (2017), 60(7), 3154-3164 SULPHURIC ACID FOR SULPHATE A SIMILAR REACTION BUT NOT SAME PAPER [link] Chemistry of Pritelivir Synthesis of pritelivir and its analogues is based on the reported methods in the literature (18−20) and presented in Figure 4. Infectious Diseases Community of Practice (2013).
A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The gene presents a complex landscape.
Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells. LOPD symptoms may present at any age.
Cowen Virtual Presentation Series – 2020 IO Next Summit at 1:15 p.m. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. EST on Monday, November 9, 2020. EST on Friday, November 13, 2020. EST ( 12:20 p.m.
It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.
NASDAQ: REGN) will webcast its presentation at the 39 th Annual J.P. The presentation is scheduled for 8:20 a.m. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. TARRYTOWN, N.Y. ,
Teva to Present Data Demonstrating UZEDY™ (risperidone) Extended-Release Injectable Suspension Significantly Prolonged Time to Impending Relapse for Adults with Schizophrenia at the American Psychiatric Association’s 2023 Annual Meeting Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. once-monthly dosing) and 2.7
Background: Endocannabinoids, which are present throughout the central nervous system (CNS), can activate CB1 and CB2 receptors. CB1 and CB2 agonists exhibit broad anti-inflammatory properties, suggesting their potential to treat inflammatory diseases. However, careful evaluation of abuse potential is necessary.
Presentations will be available for registered attendees via the Piper Sandler conference site through December 3. The Company is currently seeking FDAapproval of voclosporin for the potential treatment of LN. VICTORIA, British Columbia–( BUSINESS WIRE )– Aurinia Pharmaceuticals Inc.
FDAApproves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. This indication is approved under accelerated approval regulation based on overall response rate and duration of response. This indication is approved under accelerated approval based on overall response rate and duration of response.
Aligning cross-functional teams across the organization to ensure medical, marketing, and market access are all in lock-step is even more crucial when launching a therapy into a rare disease market. png Listing Introduction Herspiegel experts share best practices for addressing the unique aspects of successful rare disease commercialization.
Allergan, an AbbVie (NYSE: ABBV) company, today announced new data to be presented from Allergan’s leading portfolio of eye care treatments at the 2022 American Glaucoma Society (AGS) Annual Meeting being held in Nashville, TN and virtually from March 3-6. The DURYSTA data presentations coincide with the two-year anniversary of the U.S.
Eli Lilly and Company (NYSE: LLY) today announced that data from programs across its oncology portfolio and pipeline will be presented at the 2021 American Association for Cancer Research (AACR) Annual Meeting, to be held virtually April 10-15, 2021. Pipeline Highlights. Retevmo (selpercatinib). Abstract Number: CT011. Alimta (pemetrexed).
Aurinia Pharmaceuticals is a late-stage clinical biopharmaceutical company focused on developing and commercializing therapies to treat targeted patient populations that are impacted by serious diseases with a high unmet medical need. The Company’s head office is in Victoria, British Columbia and its U.S.
Metabolism of 2023 FDAApproved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. Drug Metabolism and Disposition 50 (5) 576-590; DOI: [link] [14] FDA prescribing information for palovarotene.
Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. For such trials, the US Food and Drug Administration (FDA) may allow the use of credible real-world data (RWD) and real-world evidence (RWE) in lieu of data collected in a Phase 3 trial.
Initiation of Phase III clinical trial programme for fenebrutinib, an investigational medicine designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, which may offer novel approach to suppress disease activity and slow disease progression in MS.
today announced that data from its migraine portfolio will be presented at the International Headache Congress 2021, held jointly this year by the International Headache Society and the European Headache Federation, from September 8-12. The company will share a total of 23 abstracts, including two oral presentations and one abstract lecture.
ADUHELM should be initiated in patients with mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s dementia. Food and Drug Administration (FDA) has approved an updated label for ADUHELM (aducanumab-avwa) injection 100 mg/mL solution. ADUHELM is indicated for the treatment of Alzheimer’s disease.
RAD011 is a pivotal-trial ready synthetic cannabidiol oral solution with potential utilization in multiple endocrine and metabolic orphan diseases.
Prader-Willi syndrome (“PWS”) will be the initial indication, which has been granted Orphan Drug and Fast Track Designation by the FDA.
Disease Highlights.
NYSE and TASE: TEVA),today announced six poster abstracts across its neurology portfolio will be presented at the American Academy of Neurology (AAN) Annual Meeting on April 22-27, 2023. Additional abstracts from the Enroll-HD Global Registry and AJOVY ® (fremanezumab-vfrm) will also be presented.
The findings were presented at TCT Connect , the 32nd annual scientific symposium of the Cardiovascular Research Foundation. In the first study, presented by Hemindermeet Singh, MD, of Ascension St. The symposium is chaired by William O’Neill, MD, medical director of the Center for Structural Heart Disease at Henry Ford Hospital.
– Allergan, an AbbVie (NYSE: ABBV) company, today announced that it will present new data from its leading portfolio of eye care medicines at the 2021 ASCRS (American Society for Cataract and Refractive Surgery) Annual Meeting being held July 23-27 in Las Vegas, NV. Details about Allergan’s presentations are as follows: .
Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. As your CRO partner, we deliver equally dedicated and experienced teams that understand this disease and the range of assessments used for collecting endpoint data in PAH clinical trials.
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. TARRYTOWN, N.Y. ,
Food and Drug Administration (FDA) granted Breakthrough Therapy designation for donanemab, Eli Lilly and Company’s (NYSE: LLY) investigational antibody therapy for Alzheimer’s disease (AD). Dementia due to Alzheimer’s disease is the most common form of dementia, accounting for 60 to 80 percent of all cases 1.
Biotechnology services include advising or consulting services related to the use of the above equipment, disease detection, or genealogical information, or any other service for the research, development production, analysis, detection, or provision of information including data storage and transmission related to biological materials.
These data (Presentation #DOP44) will be presented at the 17 th Congress of the European Crohn’s and Colitis Organisation (ECCO), taking place February 16-19, 2022. Additional Bristol Myers Squibb-sponsored abstracts presented at the ECCO 2022 Congress can be accessed online here. About Ulcerative Colitis.
today announced new data from its expansive neuroscience portfolio will be presented at the 2021 American Academy of Neurology (AAN) Annual Meeting, to be held virtually from April 17-22. . Key AbbVie abstracts and presentation details for the 2021 AAN Annual Meeting program are outlined below.
Teva and MedinCell Announce FDAApproval of UZEDY™ (risperidone) Extended-Release Injectable Suspension, a Long-Acting Subcutaneous Atypical Antipsychotic Injection, for the Treatment of Schizophrenia in Adults Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd.
Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and granted Priority Review for Esbriet ® (pirfenidone) for the treatment of unclassifiable interstitial lung disease (UILD). The FDA is expected to make a decision on approval by May 2021. Since its U.S. Esbriet U.S.
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