Bayer & #039;s new pharmaceutical R & D strategy to accelerate breakthrough innovations
This afternoon at 2 pm CET (8 am EDT), Bayer AG will host its Pharmaceuticals R&D Event 2023 for Investors. The company will present a deep dive on the innovation strategy in its four therapeutic core areas Oncology, Cardiovascular Diseases, Neurology & Rare Diseases and Immunology. The presentations will also include status updates on Bayer Pharmaceuticals' key assets in clinical and preclinical development as well as on its platform companies Asklepios BioPharmaceutical (AskBio), BlueRock Therapeutics (BlueRock) and Vividion Therapeutics (Vividion). (Source: World Pharma News)
Source: World Pharma News - June 28, 2023 Category: Pharmaceuticals Tags: Featured Bayer Business and Industry Source Type: news
‘ It ’ s Been a Pretty Rocky Road. ’ CDC Director Dr. Rochelle Walensky Reflects on Her Tenure During COVID-19
Dr. Rochelle Walensky became director of the U.S. Centers for Disease Control and Prevention (CDC) in January 2021, a fraught time in the country’s public health response to the COVID-19 pandemic. Americans’ confidence in the nation’s leading public health organization was at an all-time low, and Walensky had to not only manage the national response but rebuild public trust at the same time. She faced criticism over some aspects of the pandemic response, including confusing messaging and delays in processing data related to cases and severity of the disease. While most public health experts acknowledged t...
Source: TIME: Health - June 27, 2023 Category: Consumer Health News Authors: Alice Park Tags: Uncategorized COVID-19 healthscienceclimate Source Type: news
Mayo Clinic uses genomic testing broadly for rare diseases, improves patient care
NEWS RELEASE ROCHESTER, Minn. - A Mayo Clinic study published in Journal of Translational Medicine evaluated the use of genomic testing broadly for rare diseases. With the increased use of genomic testing such as multi-gene panels, exome sequencing and genome sequencing in the past decade, there is a greater opportunity to better diagnose and treat patients with rare diseases. According to the National Institutes of Health, as many as 10,000 distinct rare diseases exist and… (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - June 26, 2023 Category: Databases & Libraries Source Type: news
FDA Approves Elevidys (delandistrogene moxeparvovec-rokl) Gene Therapy to Treat Duchenne Muscular Dystrophy
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 22, 2023-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug Administration (FDA) accelerated approval of Elevidys... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - June 22, 2023 Category: Drugs & Pharmacology Source Type: news
New report shows how the Inflation Reduction Act stifles innovation for small molecule medicines
Research doesn ’t endonce a medicine is first approved by the U.S. Food and Drug Administration (FDA). Researchers often spend years after an initial approval exploring and obtaining approval of new indications for medicines to treat other patient populations desperate for new treatment options. Patients with cancer or rare diseases often depend on this post-approval research. (Source: The Catalyst)
Source: The Catalyst - June 22, 2023 Category: Pharmaceuticals Tags: Research and Development Rare Diseases Cancer Government Price Setting Source Type: news
ALS: Why this neurodegenerative disease needs 'rare' tag
Experts and medical practitioners have demanded the inclusion of the progressive neurodegenerative disease - Amyotrophic lateral sclerosis (ALS) - in the national rare disease policy to enable patients to get affordable care and treatment. The demand came ahead of the Global ALS/MND (motor neurone disease) Awareness Day, was observed on Wednesday. (Source: The Economic Times)
Source: The Economic Times - June 22, 2023 Category: Consumer Health News Source Type: news
Accelerating Rare disease Cures (ARC) Program Emerges as a Conduit for Empowering Rare Disease Stakeholders
The ARC Program strives to increase the number of treatments by promoting innovative scientific design, providing a deeper understanding of regulatory policies, and engaging with patients and their advocates, and other rare disease stakeholders. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 13, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Guidance Documents for Rare Disease Drug Development
(Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 13, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
New data show Roche ’s subcutaneously administered crovalimab achieved disease control and was well-tolerated in people with paroxysmal nocturnal haemoglobinuria (PNH)
The COMMODORE 2 study demonstrated that subcutaneous crovalimab every four weeks was non-inferior to intravenous eculizumab every two weeks, with comparable safety, in people new to C5 inhibitors1Monthly self-administration of subcutaneous crovalimab has the potential to address the high burden of a disease that requires lifelong treatment including in settings where access to currentC5 inhibitors is limited1,2The COMMODORE 1 study in people switching from currently approved C5 inhibitors supported the consistent benefit-risk profile of crovalimab as seen in the COMMODORE 2 study3Basel, 09 June 2023 - Roche (SIX: RO, ROG; ...
Source: Roche Media News - June 9, 2023 Category: Pharmaceuticals Source Type: news
New data show Roche ’s subcutaneously administered crovalimab achieved disease control and was well-tolerated in people with paroxysmal nocturnal haemoglobinuria (PNH)
The COMMODORE 2 study demonstrated that subcutaneous crovalimab every four weeks was non-inferior to intravenous eculizumab every two weeks, with comparable safety, in people new to C5 inhibitors1Monthly self-administration of subcutaneous crovalimab has the potential to address the high burden of a disease that requires lifelong treatment including in settings where access to currentC5 inhibitors is limited1,2The COMMODORE 1 study in people switching from currently approved C5 inhibitors supported the consistent benefit-risk profile of crovalimab as seen in the COMMODORE 2 study3Basel, 09 June 2023 - Roche (SIX: RO, ROG; ...
Source: Roche Investor Update - June 9, 2023 Category: Pharmaceuticals Source Type: news
Content and Format of the RDEA Meeting Request and Package | Rare Disease Endpoint Advancement Pilot Program
Content and Format of the RDEA Meeting Request | Rare Disease Endpoint Advancement Pilot Program (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 1, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Content and Format of the RDEA Proposal | Rare Disease Endpoint Advancement Pilot Program
(Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 1, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Submission Deadlines and Process | Rare Disease Endpoint Advancement Pilot Program
(Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 1, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
RDEA Pilot Program Disclosure Agreement | Rare Disease Endpoint Advancement Pilot Program
Disclosure Agreement | Rare Disease Endpoint Advancement Pilot Program (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 1, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Why the underestimated economic burden of rare diseases could be costing the U.S. trillions of dollars
Rare diseases have gained more attention in recent years, perhaps in part due to the high price tags often associated with approved therapies. But the cost of rare disease drugs is dwarfed by the cost of not having them. Our recent study shows that a lack of treatment for a rare disease is…#orphandrugtaxcredit #fda #petermarks #arc #learningandeducation #cder #nih #als #giacomochiesi #chiesigroup (Source: Reuters: Health)
Source: Reuters: Health - May 25, 2023 Category: Consumer Health News Source Type: news
