Should All U.S. Newborns Undergo Genomic Testing?
TUESDAY, May 9, 2023 -- While newborns are only screened for about 60 treatable conditions, there are hundreds of genetic disorders that have targeted treatments.
Now, a national survey of experts in rare diseases found the vast majority support DNA... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - May 9, 2023 Category: General Medicine Source Type: news
Janssen Enters Worldwide Collaboration and License Agreement with Cellular Biomedicine Group to Develop Next Generation CAR-T Therapies
HORSHAM, Pa., May 2, 2023 – Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, announced today that it has entered into a worldwide collaboration and license agreement with Cellular Biomedicine Group Inc. (CBMG) to develop, manufacture and commercialize next-generation chimeric antigen receptor (CAR) T-cell therapies for the treatment of B-cell malignancies. These investigational CD20-directed autologous CAR-Ts have demonstrated promising overall and complete response rates in Phase 1 studies in patients with relapsed/refractory non-Hodgkin’s lymphoma (NHL) in China, with the m...
Source: Johnson and Johnson - May 2, 2023 Category: Pharmaceuticals Tags: Latest News Source Type: news
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Five-year-old Seersha Sulack has a rare disease where a common cold could kill her. Now she is set to receive life-saving treatment, which had been shelved because the pharmaceutical company that once owned the license decided not to pursue approval from the US Food and Drug Administration. CNN's Elzabeth Cohen has more. (Source: CNN.com - Health)
Source: CNN.com - Health - April 27, 2023 Category: Consumer Health News Source Type: news
RDEA Pilot Program 2023 Public Workshop - 06/07/2023
The purpose of this public workshop is to discuss topics relevant to the development of endpoints for rare diseases. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - April 27, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Moderna's CEO shares what it was like to leap from pre-revenue startup to Fortune 500 company
On this week’s episode of Fortune‘s Leadership Next podcast, co-hosts Alan Murray and Michal Lev-Ram talk with Moderna CEO Stephane Bancel about drugs the company is developing to combat cancer, heart failure, and ultra-rare diseases in children; the upcoming price increase of the COVID-19…#alanmurray #michallevram #stephanebancel #deloitte #leadershipnext #moderna #flagshippioneering #noubarafeyan #noubar #rna (Source: Reuters: Health)
Source: Reuters: Health - April 26, 2023 Category: Consumer Health News Source Type: news
People warned to 'stay safe' with rare disease on the rise in the UK
In severe cases Lyme disease can cause lasting joint pain, and nerve and heart problems. (Source: Daily Express - Health)
Source: Daily Express - Health - April 24, 2023 Category: Consumer Health News Source Type: news
FDA CDER and CBER & Duke-Margolis Center for Health Policy | Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development - 06/07/2023
The purpose of this public workshop is to discuss topics relevant to the development of endpoints for rare diseases. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - April 17, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
Soligenix Provides Regulatory Update on HyBryte(TM)
PRINCETON, N.J., April 14, 2023 -- (Healthcare Sales & Marketing Network) -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where ... Biopharmaceuticals, Oncology, FDA Soligenix, HyBryte, synthetic hypericin sodium, CTCL (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 14, 2023 Category: Pharmaceuticals Source Type: news
Citizen Scientists Are Driving Medical Research. Now They Need A Constitution
More than a dozen people showed up in November, 2007, at the National Institutes of Health (NIH) lab run by Christopher Austin. The parents of children dying from the lethal cholesterol metabolism disorder, Niemann-Pick disease type C (NPC), gathered alongside scientists and doctors looking for a treatment. They wanted to work together to find effective drugs and get them into clinical trials within a few years, accelerating the typical timeline for drug development. They hoped to find a compound that might extend the lives of children with the rare disease, who typically died by age 19.
In terms of the science, the meetin...
Source: TIME: Health - April 14, 2023 Category: Consumer Health News Authors: Amy Dockser Marcus Tags: Uncategorized freelance health Source Type: news
New Drug May Treat Rare Diseases That Make Exposure to Sunlight Painful
THURSDAY, April 13, 2023 -- It sounds like the stuff of a vampire novel, but for people with a group of rare genetic disorders, exposure to sunlight can cause excruciating pain.
Now, an experimental medication is showing promise for helping them... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - April 13, 2023 Category: General Medicine Source Type: news
Health Highlights: April 13, 2023
Federal appeals court keeps abortion pill available temporarily. However, the ruling blocks getting mifepristone through the mail and rolls back other steps the government had taken to improve access. Read more
New drug may treat rare diseases that... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - April 13, 2023 Category: General Medicine Source Type: news
New Drug, Dersimelagon. May Treat Rare Diseases That Make Exposure to Sunlight Painful
THURSDAY, April 13, 2023 -- It sounds like the stuff of a vampire novel, but for people with a group of rare genetic disorders, exposure to sunlight can cause excruciating pain.
Now, an experimental medication is showing promise for helping them... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - April 13, 2023 Category: General Medicine Source Type: news
From Lab to Living Room: mRNA Therapeutics
In recent years, advancements in biotechnology have led to the development of innovative medicines such as messenger RNA (mRNA) therapeutics: a mysterious new field of science with tremendous applications in cancer, rare disease, and infectious disease. Yet, the complex science behind these novel…#rna #andrewberg #arpagaray #moderna #merck #paulburton #phd #fda #pameladavis #svpofmedicalaffairs (Source: Reuters: Health)
Source: Reuters: Health - April 7, 2023 Category: Consumer Health News Source Type: news
Lab Notes: CHOP gets $10M research gift; Vallon seeks greater shareholder support for merger
News from the Philadelphia-area life sciences industry this week includes a biopharmaceutical firm seeking greater shareholder approval for its proposed merger, a medical device developer signing a debt financing agreement for up to $60 million, and a pediatric care provider receiving over $10 million for rare disease research. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 7, 2023 Category: Pharmaceuticals Authors: John George Source Type: news
MedPAC proposal threatens access to medicines in Part B
Next week, the Medicare Payment Advisory Commission (MedPAC) will vote on a number of policy proposals for changing Medicare Part B. One of the proposals would recommend that Congress adjust the payment for Part B medicines withaccelerated approval, including medicines for serious and life-threatening diseases like cancers, rare diseases and HIV. This could compromise patient access to medicines, which is the opposite of what accelerated approval is designed to do. Here are three reasons why MedPAC should vote no on this proposal. (Source: The Catalyst)
Source: The Catalyst - April 6, 2023 Category: Pharmaceuticals Tags: Medicare Part B Accelerated Approval Source Type: news