Rare Diseases Videos
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Tonix Pharmaceuticals CEO talks through 2021 highlights, ending year with $179M in cash - Video
Tonix Pharmaceuticals Holding Corp. CEO Seth Lederman talks to Proactive about the New Jersey-based clinical-stage biopharmaceutical company's 2021 highlights. Lederman says the group ended the year with $179 million in cash and cash equivalents, entering 2022 'poised for growth.' Lederman highlights the FDA's recent award of Orphan-Drug designation for Tonix's TNX-2900 to treat Prader Willi ...
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The Best Method to Relieve Pain at Home - LONGEST MStim Reha LGT-231 - Video
LONGEST MStim Reha LGT-231 is a portable FDA-cleared TENS + NMES unit for individuals to relieve pain easily and effectively at home. It is easy to use with preset protocols and clear body navigation. The device can be used to relieve pain caused by arthritis, sports injuries, multiple sclerosis (MS), fibromyalgia, and spinal cord injury, as well as period pain, pelvic pain, and low back ...
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Precious Baby Project - Video
One photographer is helping make special memories for parents of children with rare diseases. “I hope to show the world that babies – and people - with special needs are beautiful and valuable.” ...
By CSL Behring
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ARM`s 2022 Cell & Gene State of the Industry Briefing - Video
The Alliance for Regenerative Medicine’s annual Cell & Gene State of the Industry Briefing, a top-level gathering for key stakeholders in the field. This webinar includes ARM’s annual yearly update on the sector’s progress, followed by two panel sessions featuring top CEOs and executives highlighting future inflection points for cell and gene therapies in the coming year. ...
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Jeeva eClinical Cloud - Scalable Modular SaaS for Clinical Trials in Real Life - Video
Paperless | Contact-free | Clinical Research Jeeva is a premium eClinical cloud solution that helps you reach your recruitment and retention targets sooner. Clinical researchers can pre-screen participants, obtain informed consent, enroll subjects, and begin their studies faster by switching from traditional clinical studies to decentralized, remote, and hybrid studies like Jeeva. When you ...
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Baebies SEEKER | Workflow - Video
Watch the lab workflow of SEEKER, an innovative FDA authorized and CE marked newborn screening platform for lysosomal storage disorders. Learn more: https://www.baebies.com/SEEKER At Baebies, we believe that everyone deserves a healthy ...
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GenBio - Genome Biologics: Our Technology - Vdeo
Modern drug discovery is inefficient, expensive and risky. On average, there are 2000 failed compounds sitting on pharmaceutical companies shelves, and this number grows at the rate 200 drugs per year. Furthermore, the average costs of a successful drug introduction is a staggering $3bln with an average time-to-market of more than 10 years. This is due to extensive discovery costs including: R&D, ...
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A patient journey vol.1: Living with a rare disease - Video
In this video, you will learn more about Philipp’s rare disease and how it has affected his life so far. “Rare", however, is actually not that rare ...
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Finding Tomorrows: Full Film - Video
20 years of rare opportunities. 20 years of personal impact. Despite being faced with potential demise during the company’s infancy, BioMarin persevered to become one of the world leaders in developing therapies for rare genetic diseases. Watch the 20 year journey of the people of BioMarin finding tomorrows by staying focused on the science to make a big difference for small patient ...
By BioMarin
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BioMarin’s Scientific Motivation - Video
At BioMarin we are tackling scientific problems that no one has worked on before and we are paving the way to develop new scientific discoveries. Learn more from our employees and why they enjoy working at BioMarin. BioMarin is a global biotechnology company focused on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and ...
By BioMarin
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BioMarin’s Connection to Patients - Video
At BioMarin, the connection and strong relationships we have with our patients energizes our employees and our company culture every day. Together, we aspire to positively impact the lives of those living with rare diseases. ...
By BioMarin
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What is MPS IVA? - Video
MPS IVA, also known as Morquio A Syndrome, is a rare disease that affects major organs in the body. Learn more about Morquio A Syndrome from the ...
By BioMarin
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What is CLN2? - Video
CLN2 is an ultra-rare and rapidly progressing pediatric brain disorder and one of the most common forms of a group of inherited disorders collectively known as Batten disease. Learn more about CLN2 disease from parents with children who have CNL2 disease. ...
By BioMarin
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