Syndrome

Sentynl Therapeutics Announces Global Acquisition of Zokinvy® (Lonafarnib) for Treatment of Hutchinson-Gilford Progeria Syndrome from Eiger BioPharmaceuticals

Retrieved on: 
Friday, May 3, 2024
Sale, FDA, Mortality, Bankruptcy, Progeria Research Foundation, Progeria, Hearing, Zydus Lifesciences, Food, Lonafarnib, OTC, PRF, HGPS, Partnership, Doctor of Philosophy, Collection, Research, European, Disease, Patient, Eiger, United States bankruptcy court, Acquisition, PALO, DSM-IV codes, MBA, Therapy, Syndrome, PDPL, Pharmaceutical industry

SOLANA BEACH, Calif. and AHMEDABAD, India and PALO ALTO, Calif., May 3, 2024 /PRNewswire/ -- Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (Zydus Group), and Eiger BioPharmaceuticals, Inc. (OTC: EIGRQ), a commercial-stage biopharmaceutical company focused on the development of innovative therapies for rare metabolic diseases, today announced the closing of the sale of Eiger's Zokinvy® (lonafarnib) program to Sentynl.

Key Points: 
  • Collectively known as progeria, HGPS and PDPL are ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients.
  • Since we first launched PRF in 1999, we have achieved tremendous progress in global awareness, breakthrough research, and treatment of progeria.
  • Under the terms of the acquisition, Sentynl acquired global rights to Zokinvy and will be responsible for its manufacture and commercialization.
  • For questions on continued access to Zokinvy, please contact the Sentynl Cares support team at 1-888-251-2800 Monday-Friday, 8 am-8 pm ET.

May is International 5p- Syndrome (Cri du Chat) Syndrome Awareness Month

Retrieved on: 
Friday, May 3, 2024
Education, Society, International, Syndrome, Social media, Perseverance, Happiness, Life, Genetic testing, Patient, Campaign, Chromosome, Quality of life, Drink, Organization, Disability

LAKEWOOD, Calif., May 2, 2024 /PRNewswire-PRWeb/ -- The 5p- Society of North America, along with families and support organizations from over 95 countries, are bringing awareness of 5p Deletion Syndrome also known as 5p Minus (5p-) Syndrome. Over sixty (60) years ago, Dr. Jerome Lejeune discovered this genetic condition by noticing a similar characteristic in several patients that he had been observing of a high pitched "cat-like sound" vocalization. After doing genetic testing on these patients, he realized the connection is caused by a permanent deletion on the "p" arm of the 5th chromosome. The 2024 awareness campaign celebrates these unique individuals by raising awareness around the world through education and community events, such the "Faces of 5p-"campaign, our "Stripy Socks" campaign, as well as a "Virtual 5K for 5p-" event and family regional gatherings.

Key Points: 
  • Celebrating the amazing and unique individuals with 5p- Syndrome throughout the month of May by spreading awareness around the world.
  • LAKEWOOD, Calif., May 2, 2024 /PRNewswire-PRWeb/ -- The 5p- Society of North America, along with families and support organizations from over 95 countries, are bringing awareness of 5p Deletion Syndrome also known as 5p Minus (5p-) Syndrome.
  • In an ongoing effort to raise awareness, members and supporters are encouraged to participate in events throughout the month.
  • * Virtual 5k for 5p-, to raise awareness and money for programs that benefit the 5p Deletion Syndrome community.

PIONEERING CHICAGO PHYSICIAN TREATS HIS 1,000th R-CPD (RETROGRADE CRICOPHARYNGEUS DYSFUNCTION) PATIENT

Retrieved on: 
Wednesday, May 1, 2024
Diagnosis, Patient, Collection, Syndrome, Physician, Bloating

DOWNERS GROVE, Ill., May 1, 2024 /PRNewswire/ -- Dr. Robert Bastian, renowned laryngologist and founder of Bastian Voice Institute (BVI), achieves a major milestone on May 1, 2024. Kicking off National Digestive Diseases Awareness Month, Dr. Bastian will treat his 1,000th patient with the diagnosis of R-CPD (Retrograde Cricopharyngeus Dysfunction), a digestive disorder that he first described as a detailed syndrome in 2015, when he also became the first to successfully treat and resolve this condition. R-CPD occurs when the upper esophageal sphincter will not relax in order to release intestinal gas and is the formal name for a condition comprised by a collection of symptoms all tied to an inability to burp (or burp sufficiently).    

Key Points: 
  • "The significance of treating my 1,000th R-CPD patient during National Digestive Diseases Awareness Month couldn't be more fitting," states Bastian.
  • These patients have also many times been treated for the individual symptoms, rather than the underlying cause, resulting in little to no relief.
  • Bringing awareness to this specific condition, and shedding light on the readily available, proven treatment is of paramount importance to me."
  • Until Dr. Bastian codified R-CPD as a condition, it was unknown as a specific diagnosis in the medical community.

Corcept Announces Positive Results From Open-Label Portion of Pivotal Phase 3 GRACE Trial in Patients With Cushing’s Syndrome

Retrieved on: 
Monday, April 22, 2024
GRACE, Hyperglycemia, University, Patient, Syndrome, SBP, Adrenal insufficiency, Safety, NDA, Waist, Ann Arbor, Michigan, MD, Doctor of Philosophy, Vaginal bleeding, DSM-IV codes, Doctor of Pharmacy, Diabetes, Hypertension, Metabolism, Internal medicine, Endocrinology, Division, Deficit spending, DBP, Pharmaceutical industry

In the “open-label” phase, 152 patients with Cushing’s syndrome and either hypertension, hyperglycemia or both received relacorilant for 22 weeks.

Key Points: 
  • In the “open-label” phase, 152 patients with Cushing’s syndrome and either hypertension, hyperglycemia or both received relacorilant for 22 weeks.
  • GRACE’s primary endpoint is maintenance of blood pressure control in the “randomized withdrawal” phase, with maintenance of glycemic control as the key secondary endpoint.
  • Other key secondary and exploratory endpoints in the randomized withdrawal phase include changes in weight, waist circumference, cognitive impairment and Cushing’s Quality of Life score.
  • Patients in the open-label phase exhibited clinically meaningful and statistically significant improvements in hypertension, hyperglycemia and other key secondary and exploratory endpoints.

FSIS ISSUES PUBLIC HEALTH ALERT FOR GROUND BEEF PRODUCTS DUE TO POSSIBLE E. COLI O157:H7 CONTAMINATION

Retrieved on: 
Saturday, April 20, 2024
Food safety, Syndrome, Person, Poultry, Dysentery, Independence Avenue, Commerce, Kidney failure, USDA, Human, H7, Dehydration, HUS, TDD, Pallor, Meat, Temperature, Chronic pain, FSIS, Bruise, Office for Civil Rights, Civil and political rights, Poultry farming

WASHINGTON, April 20, 2024 – The U.S. Department of Agriculture’s Food Safety and Inspection Service (FSIS) is issuing a public health alert for ground beef products that may be contaminated with E. coli O157:H7.

Key Points: 
  • WASHINGTON, April 20, 2024 – The U.S. Department of Agriculture’s Food Safety and Inspection Service (FSIS) is issuing a public health alert for ground beef products that may be contaminated with E. coli O157:H7.
  • FSIS is issuing this public health alert to ensure that consumers are aware that these products should not be consumed.
  • The products have a “Use/Freeze by” date of April 22, 2024, and packaging date of “032824.” The list of products that are subject to the public health alert can be found here .
  • The company notified FSIS that they inadvertently used a portion of the contaminated beef to produce ground beef products that they subsequently shipped into commerce.

TScan Therapeutics Provides Clinical Pipeline Update and Highlights Near-Term Priorities

Retrieved on: 
Tuesday, April 16, 2024
Patient, Cancer, T-ALL, MRD, HLA, Collection, Trial of the century, HCT, AML, IND, Blood, Syndrome, Acute myeloid leukemia, Acute leukemia, Neoplasm, TCR, Therapy, MDS, ALL, Medical imaging

WALTHAM, Mass., April 16, 2024 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies (TCR-T) for the treatment of patients with cancer, today provided an update on its solid tumor and heme malignancies clinical programs.

Key Points: 
  • “We continue to make meaningful progress across both our solid tumor and heme malignancies Phase 1 clinical programs.
  • This should allow for rapid enrollment into the treatment protocol over the course of the year,” said Gavin MacBeath, Ph.D., Chief Executive Officer.
  • Phase 1 solid tumor clinical study has been initiated; first three patients expected to be dosed in early May 2024.
  • Completion of Phase 1 enrollment and reporting of one-year clinical and translational data on initial patients is anticipated in the second half of 2024.

Neurogene Announces Upcoming Presentation of Safety Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome at ASGCT Meeting

Retrieved on: 
Monday, April 22, 2024
Research, Neurology, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Other Science, Science, Brain, MECP2, Safety, Neurological disorder, Rett syndrome, Cell, Patient, Gene, Society, DSM-IV codes, Inc., Clinical trial, Annual general meeting, Syndrome, Pharmaceutical industry

Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome will be presented at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting.

Key Points: 
  • Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome will be presented at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting.
  • The data show that the NGN-401 gene therapy candidate has been generally well-tolerated, and there have been no treatment-emergent or procedure-related serious adverse events or signs of MeCP2 overexpression-related toxicity observed in any patient, including a patient with a mild variant predicted to result in residual MeCP2 function.
  • “Rett syndrome is a complex neurological disorder with a narrow therapeutic window, and conventional gene therapy approaches have been unable to provide therapeutic protein levels without detrimental overexpression.
  • Therefore, we believe it is important to share this safety update at the ASGCT Meeting, in advance of our expected fourth quarter 2024 interim efficacy read-out, as we have multiple months of data from three patients showing NGN-401 has been generally well-tolerated.”
    Title: Preliminary Safety Results from the Ph1/2 study of NGN-401, a Novel Regulated Gene Therapy for Rett Syndrome

Fullscript Gathers Industry Leaders in Proactive, Whole Person Care for Cardiometabolic Summit

Retrieved on: 
Monday, April 15, 2024
Cardiology, Practice Management, Telemedicine, Virtual Medicine, Pharmaceutical, Health, Health Technology, Vitamins, Supplements, Fitness & Nutrition, Education, CVD, Cardiovascular disease, Women's health, Conditional sentence, MD, Patient, Doctor of Philosophy, Risk, Syndrome, Nursing

Fullscript , a leader in healthcare platforms for whole person care, today announced that on April 17-18th, 2024, it will be hosting a mixed audience of conventional and functional clinicians for a virtual Cardiometabolic Summit .

Key Points: 
  • Fullscript , a leader in healthcare platforms for whole person care, today announced that on April 17-18th, 2024, it will be hosting a mixed audience of conventional and functional clinicians for a virtual Cardiometabolic Summit .
  • Fullscript’s Cardiometabolic Summit will bring together thousands of cross-functional providers and primary care clinicians across various disciplines as they dive deep into trending topics about cardiometabolic care and explore ways to help patients prevent and manage chronic illness as part of a wider look at whole person health.
  • “At Fullscript, we are dedicated to scaling the delivery of whole person care by helping providers drive proven, better health outcomes.
  • “As clinicians, we have a responsibility to better understand the ways in which providers and patients alike can engage in whole person healthcare,” said Jeff Gladd, MD, chief medical officer, Fullscript.

Innate Pharma Announces Advancement of Sanofi-developed NK Cell Engager SAR443579 / IPH6101 Progressing to Phase 2 for Blood Cancer Patients

Retrieved on: 
Monday, April 15, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Cancer, Safety, Hematology, IPHA, Research, AML, IPH, Euronext Paris, Fast track (FDA), Acute lymphoblastic leukemia, Patient, Society, Bone marrow, Syndrome, Acute myeloid leukemia, Clinical trial, Pharmaceutical industry

SAR443579 is an investigational trifunctional anti-CD123 NKp46xCD16 NK cell engager from a joint research collaboration between Innate Pharma and Sanofi.

Key Points: 
  • SAR443579 is an investigational trifunctional anti-CD123 NKp46xCD16 NK cell engager from a joint research collaboration between Innate Pharma and Sanofi.
  • SAR443579 received FDA Fast Track Designation for the treatment of acute myeloid leukemia.
  • “The progression of SAR443579 to the Phase 2 expansion part of the clinical trial in blood cancers is another step in bringing this innovative NK cell engager to patients,” said Dr. Sonia Quaratino, Chief Medical Officer of Innate Pharma.
  • “SAR443579 has shown promising clinical efficacy in the dose escalation of the Phase 1/2 in R/R AML patients, and we look forward to the dose expansion part of the study."

Toyota Research Institute Announces Multimillion-Dollar Challenge to Accelerate Research in New Advanced Materials

Retrieved on: 
Thursday, April 18, 2024
EVS, Toyota, Research, Artificial intelligence, EV, Medium, LOS, TRI, Woven Planet Holdings, Syndrome, Energy

LOS ALTOS, Calif., and CAMBRIDGE, Mass., April 18, 2024 /PRNewswire/ -- Toyota Research Institute (TRI) announces a multiyear, multimillion-dollar challenge aimed at closing the gap between recent advances in AI prediction of new materials and finding the actual "recipe" needed to make these hypothetical materials in the real world. 

Key Points: 
  • While this is encouraging, a bottleneck occurs when trying to create these materials in the laboratory.
  • Developing the recipe to create even a single new material in the lab – the act of synthesis – can take years.
  • "Accelerating the synthesis of computer-predicted materials could be a game changer in the development of advanced technologies such as EV batteries," said Brian Storey , TRI's Senior Director of Energy & Materials.
  • "Our intention is to shave years off the new materials discovery process by creating a collaborative research challenge amongst academics and research partners around the world."