Rare disease

ORYZON Selected as Associated Partner in the First Important Project of Common European Interest (IPCEI) in the Health Sector (Med4Cure Project)

Retrieved on: 
Mercoledì, Maggio 29, 2024
Consortium, Commission, European Commission, Omnicom Group, CDTI, Recovery, Research, Government, Communication, Genomics, Rare disease, Personalized medicine

The Member States will provide up to €1 billion in public funding, which is expected to unlock an additional €5.9 billion in private investments.

Key Points: 
  • The Member States will provide up to €1 billion in public funding, which is expected to unlock an additional €5.9 billion in private investments.
  • Oryzon has been selected to participate as an Associated Partner in the Consortium.
  • The Med4Cure is a macro-project that deploys 14 scientific projects to be developed by 13 companies as Direct Partners and 11 as Associated Partners.
  • The project to be developed by Oryzon is called VANDAM and aims to validate epigenetic experimental drugs by applying a personalized medicine approach for rare and orphan diseases.

Rocket Pharmaceuticals Reports First Quarter 2024 Financial Results and Highlights Recent Progress

Retrieved on: 
Lunedì, Maggio 6, 2024
Research, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, Oncology, Education, FDA, CFO, Disorder, Regenerative medicine, University of California, Alexion Pharmaceuticals, PDUFA, Arena Pharmaceuticals, Danon disease, European Medicines Agency, Strategic planning, UCLA, Food, Molecule, Stanford University, Empire, BLA, Medication, Corporate communication, Definitive, Fanconi anemia, Patient, Increment, Safety, Rare disease, Regeneron Pharmaceuticals, AAV, Immunology, EMA, FA, Division, PKD, University, Marketing, MAA, Cell, Biologics license application, Pharmacology, Rare Disease Day, Disease, Mirati Therapeutics, Haematopoietic system, Gene, Society, Pyruvate kinase deficiency, Liberty Science Center, Annual general meeting, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the quarter ending March 31, 2024.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the quarter ending March 31, 2024.
  • In April, Rocket announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102 for the treatment of FA.
  • MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial.
  • Mr. Ondrey was previously the CFO at Mirati Therapeutics and has held multiple senior finance leadership positions at Arena Pharmaceuticals, Alexion Pharmaceuticals, and Regeneron Pharmaceuticals.

National Organization for Rare Disorders (NORD) Announces 2024 Rare Impact Award Honorees, Emmy Award-Winning Journalist, Peter Alexander to Host

Retrieved on: 
Lunedì, Aprile 22, 2024
Education, FDA, Haemophilia, Risk, Genetics, Policy, Legislation, Louisiana House of Representatives, Diagnosis, Community, Enzyme replacement therapy, DSM-IV codes, Universal Pictures, Usher syndrome, NBC News, Primary immunodeficiency, Metabolic disorder, Therapy, Abbey, Coagulopathy, IgA nephropathy, Hospital, Rare disease, BioMarin Pharmaceutical, Proteinuria, Alpha-mannosidosis, Regeneron Pharmaceuticals, House, Immunoglobulin A, Medicine, Biogen, Universal Studios Hollywood, HFSC, PDT, APDS, Friedreich's ataxia, Disease, Science, Patient, Caregiver, Representative, Activated, Pharmaceutical industry

"Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."

Key Points: 
  • "Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."
  • "Having witnessed my sister's journey, I know the challenges rare disease families face are not just physical but also emotional, mental, and financial.
  • In addition to the individual award recipients, the Rare Impact Awards are also honoring companies that have developed treatments changing the lives of those with rare diseases.
  • Regeneron Pharmaceuticals for Veopoz™: The first and only treatment indicated specifically for CHAPLE disease, a rare hereditary immune disease.

Acadia Appoints Elizabeth H. Z. Thompson, Ph.D. as Executive Vice President, Head of Research and Development

Retrieved on: 
Martedì, Aprile 2, 2024
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Mental Health, Clinical Trials, AbbVie, Prader–Willi syndrome, Scripps Research, IGM, Horizon, Delusion, Executive, Harvey Mudd College, Patient, Acquisition, Rett syndrome, Doctor of Philosophy, Myenteric plexus, Chemistry, Research and development, Hallucination, Rare disease, Amgen, ACAD, FDA, Pharmaceutical industry

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the appointment of Dr. Elizabeth H. Z. Thompson as Executive Vice President, Head of Research and Development.

Key Points: 
  • Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the appointment of Dr. Elizabeth H. Z. Thompson as Executive Vice President, Head of Research and Development.
  • Dr. Thompson will lead research and development at Acadia and serve as a member of the company’s Executive Leadership Team, reporting to Steve Davis, Chief Executive Officer of Acadia.
  • “Liz is a preeminent drug research and development leader with extensive experience in the advancement and commercialization of first in class therapies,” said Steve Davis, Chief Executive Officer.
  • She joined Horizon in 2018 and served in roles of increasing responsibility that culminated at Executive Vice President, Research and Development.

Global Immune Thrombocytopenia Drug Market Report 2024 with Analysts' Recommendations - Collaboration Initiatives with Healthcare Providers and Amplify Development of IV Drugs for Chronic Applications - ResearchAndMarkets.com

Retrieved on: 
Martedì, Marzo 12, 2024
Biotechnology, Pharmaceutical, Health, Hospital, TPO, Diagnosis, Research, PCR, Flow cytometry, Prevalence, Genetic testing, Disease, Porter's five forces analysis, Drug discovery, Incidence, Patient, SWOT, ITP, Autoantibody, Administration, Treatment, Rare disease, Drug development, Safety, Thrombocytopenia, Mimetic theory, USD, Marketing, Polymerase chain reaction, Ageing, Therapy, Pharmaceutical industry

The Global Immune Thrombocytopenia Drug market showcased growth at a CAGR of 21.32% during 2019-2022.

Key Points: 
  • The Global Immune Thrombocytopenia Drug market showcased growth at a CAGR of 21.32% during 2019-2022.
  • Immune Thrombocytopenia Drug Market is being propelled by rising prevalence of immune thrombocytopenia worldwide is a significant driver of the ITP drug market.
  • Technological advancements drive innovation and progress in the global Immune Thrombocytopenia Drug Market, accelerating drug discovery, development, manufacturing, and clinical practice.
  • The report analyses the Immune Thrombocytopenia Drug Market by End-User (Hospitals and Clinics, Specialty Centers, Research and Academic Institutes).

Alexion Canada Appoints Karen Heim as New General Manager

Retrieved on: 
Martedì, Aprile 2, 2024
Circulatory system, Neurology, AstraZeneca, GSK plc, Metabolism, Hematology, Cardiology, Patient, DSM-IV codes, Research, Alexion Pharmaceuticals, Bachelor of Science, Acquisition, University, Senior, Metallurgy, Nephrology, Queen's University, Rare disease, Immunity (medical), Alexion, Ophthalmology, Pharmaceutical industry

MISSISSAUGA, ON, April 2, 2024 /CNW/ - Alexion Pharma Canada Corp., AstraZeneca's Rare Disease group, is pleased to announce the appointment of Karen Heim as Vice President and General Manager, Canada, effective April 1st, 2024.

Key Points: 
  • MISSISSAUGA, ON, April 2, 2024 /CNW/ - Alexion Pharma Canada Corp., AstraZeneca's Rare Disease group, is pleased to announce the appointment of Karen Heim as Vice President and General Manager, Canada, effective April 1st, 2024.
  • In this role, Ms. Heim will lead the Canadian affiliate in its unwavering commitment to helping people affected by rare diseases live longer, healthier lives.
  • "Karen's deep healthcare experience and incredible passion make her the perfect fit to lead this mission in Canada and drive it with expertise and ambition."
  • "Taking on this leadership role at Alexion Canada as it continues to grow, with a focus on research and innovation, is incredibly exciting," says Ms. Heim.

Engaging Patient Communities: A Pathway to Clinical Trial Success, Upcoming Webinar Hosted by Xtalks

Retrieved on: 
Martedì, Marzo 26, 2024
Trust, Marketing, Black Women's Health Imperative, Clinical trial, Rare disease, Communication, Human, Nursing

TORONTO, March 26, 2024 /PRNewswire-PRWeb/ -- In this webinar, the expert speakers will focus on the critical role of patient communities in advancing clinical trials. They will explore best practices for engaging with patient communities to enhance recruitment, retention and overall success of the trial. Based on case studies and interactive discussions, attendees will learn how to build trust, foster communication and address the unique needs of patient communities.

Key Points: 
  • The featured speakers will discuss how to capture the patient voice for clinical trials.
  • TORONTO, March 26, 2024 /PRNewswire-PRWeb/ -- In this webinar, the expert speakers will focus on the critical role of patient communities in advancing clinical trials.
  • They will explore best practices for engaging with patient communities to enhance recruitment, retention and overall success of the trial.
  • Register for this webinar today to discover strategies for meaningful collaboration, which in turn can drive innovation and improve the clinical trial experience.

Rare Disease Diversity Coalition Honors Champions and Unveils Resources at 2nd Annual Rare Disease Day Reception

Retrieved on: 
Giovedì, Marzo 14, 2024
Novartis, Organization, Barrier, Disease, Health equity, Therapy, Rare disease, Vertex Pharmaceuticals, FDA, Pharmaceutical industry

WASHINGTON, March 14, 2024 /PRNewswire/ -- The Rare Disease Diversity Coalition (RDDC) proudly hosted its 2nd Annual Rare Disease Day Reception and 1st Annual RISE (Resilient, Impassioned, Strong, Empowered) AWARDS on February 28, 2024, at Long View Gallery in Washington, DC.

Key Points: 
  • WASHINGTON, March 14, 2024 /PRNewswire/ -- The Rare Disease Diversity Coalition (RDDC) proudly hosted its 2nd Annual Rare Disease Day Reception and 1st Annual RISE (Resilient, Impassioned, Strong, Empowered) AWARDS on February 28, 2024, at Long View Gallery in Washington, DC.
  • The event, attended by a larger audience than last year, demonstrated the growing momentum within the rare disease community.
  • The highlight of the evening was the awards ceremony, which honored nine outstanding contributors to the rare disease community, including individuals, organizations, and legislative champions.
  • Looking ahead, RDDC is energized by the support, stories, and successes shared at this year's Rare Disease Day Reception.

Zenith Epigenetics Proudly Supports Rare Disease Day

Retrieved on: 
Giovedì, Febbraio 29, 2024
Rare disease, Head, NUT, Aggression, Neck, Quality of life, Health, Zenith, Cell, Prevalence, Prognosis, Patient, Nervous tissue, Clinical trial, NUT carcinoma, Disease, Survival, Sympathetic nervous system, Diagnosis, Metastasis, BET, Carcinoma, Cancer, Pharmaceutical industry

ZEN-3694 Expands Development to Include Multiple Rare Oncology Indications

Key Points: 
  • Calgary, Alberta--(Newsfile Corp. - February 29, 2024) - Zenith Epigenetics Ltd. ("Zenith" or the "Company") announces its support of Rare Disease Day by highlighting our expanded development efforts of BET inhibitor ZEN-3694 for rare oncology diseases.
  • Rare Disease Day takes place every year on the last day of February, with this leap year day being the rarest of all Rare Disease Days.
  • This event is a global movement to promote equity in healthcare, and access to diagnosis and therapies for individuals living with rare diseases.
  • We are very pleased that NUT carcinoma patients receiving ZEN-3694 in clinical trial and compassionate use settings have benefitted from our drug," said Donald McCaffrey, CEO of Zenith Epigenetics.

Early Pulmonary Fibrosis Detection Holds the Key to Better Health Outcomes for Canadians Living with COVID-19 and Rheumatoid Arthritis

Retrieved on: 
Mercoledì, Febbraio 28, 2024
Rare disease, Oxygen, Connective tissue disease, Research, Scleroderma, Physician, St. Joseph's Health Centre, Lupus, Crackle, Myositis, Pulmonary fibrosis, Life, CPFF, COVID-19, Signs, Rheumatoid arthritis, COVID, Patient, Disease, Diagnosis, Connective tissue, Interview, Face, Pharmaceutical industry

Canadians who have had COVID-19, and other chronic diseases, are experiencing Pulmonary Fibrosis (PF) at significantly higher rates than others, seemingly providing a causal link that researchers are investigating.

Key Points: 
  • Research is shedding light on the growing rate of pulmonary fibrosis (PF) being found in people suffering from long-term COVID-19.
  • One study, Post COVID-19 pulmonary fibrosis; a meta-analysis study , found that almost 45% (44.9%) of study participants began suffering from PF after contracting COVID-19.
  • Canadians suffering from long COVID are encouraged to watch for the early signs of pulmonary fibrosis, a disease that is growing in this population.
  • Dr. Janet Pope's webinar includes explanations for both physicians and thousands of people living with connective tissue diseases of what pulmonary fibrosis symptoms to watch for.