Rare disease

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

Retrieved on: 
Tuesday, April 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: 
Tuesday, March 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: 
Tuesday, April 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: 
Tuesday, March 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: 
Thursday, March 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: 
Thursday, February 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: 
Wednesday, February 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.

Recursion Hosts Second Annual Rare Disease Day Events for Utah’s Rare Disease Community

Retrieved on: 
Friday, March 1, 2024
Rare disease, RUN, Intelligence, Drug discovery, Nature, Hope, Entrepreneurship, ARUP Laboratories, University, Organization, Patient, Disease, Diagnosis, Biology, RDAC, RARE, Recursion, Laboratory, Partnership, Pharmaceutical industry

An estimated one in 10 people in the United States have a rare disease.

Key Points: 
  • An estimated one in 10 people in the United States have a rare disease.
  • The event recognized advocates who have been instrumental in building Utah’s rare disease community and have dedicated themselves to support this important work.
  • “There is an urgency for diagnoses, treatment and cures for the rare disease community,” said Gina Zanik, co-founder and executive director of Rare and Undiagnosed Network and Vice Chair of the Utah Rare Disease Advisory Council.
  • Rare disease patients came together at Recursion with the RARE and Undiagnosed Network (RUN), RARE-X , BioHive , ARUP Laboratories , BioUtah , the University of Utah and the Rare Disease Advisory Council (RDAC) for the Rare Disease Day event.

CENTOGENE Collaborates on Research Published in Science Showing Immunopathological Landscape of Human Pre-TCRα Deficiency

Retrieved on: 
Thursday, February 29, 2024
Infection, Rare disease, Senior, Genomics, Research, Vertebrate, Penetrance, Prevalence, CNTG, Autoimmunity, Health, Rare Disease Day, Patient, Genetics, Immunity, Immunology, Human, Vaccine

The study revealed that complete pre-TCRα deficiency is rare in humans and less severe than anticipated.

Key Points: 
  • The study revealed that complete pre-TCRα deficiency is rare in humans and less severe than anticipated.
  • Prof. Peter Bauer, Chief Medical and Genomic Officer at CENTOGENE, said, "This research advances our understanding of pre-TCRα deficiency significantly.
  • In analyzing genomic and phenomic data, CENTOGENE researchers helped establish the association between partial pre-TCRα deficiency and autoimmunity, with a higher prevalence than initially expected.
  • Christian Beetz, Senior Director Genomic Innovation at CENTOGENE, added, “Until now, the impact of pre-TCRα deficiency has been largely unknown.

PRISM MarketView Highlights Work of Emerging Companies on Rare Disease Day

Retrieved on: 
Thursday, February 29, 2024
Rare disease, Therapy, Regenerative Medicine Advanced Therapy, FAP, Concha bullosa, Sulindac, Chemistry, Fast track (FDA), Muscular dystrophy, FDA, Sensation, News, Eflornithine, DMD, Prader–Willi syndrome, BLA, Chromosome, RDEB, PDUFA, Skin, Voyager, PRISM, Duchenne muscular dystrophy, Safety, Neurocrine Biosciences, Rare Disease Day, Genetics, Fatigue, Walking, PWS, Patient, DSM-IV codes, Medicine, Disease, Exosome, FA, CMC, Priority review, Pharmaceutical industry

All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.

Key Points: 
  • All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.
  • Today on Rare Disease Day, PRISM MarketView highlights emerging companies working to deliver life changing treatments for those living with rare diseases.
  • RDEB is a severe rare disease characterized by extremely fragile, and extensive blistering and wounds.
  • Capricor is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases.