Government-funded initiatives such as the Cancer Moonshot support not only the primary science that often serves as a foundation for medical advances, but also ensure that patients with rare cancers are not overlooked.
To help clinicians diagnose rare disease more quickly and accurately, many healthcare organizations are embracing technology solutions like natural language processing (NLP) tools that can create augmented intelligence workflows that facilitate the rapid search of unstructured clinical data from multiple data sources.
As technology advances, AI-powered tools will increasingly reduce the administrative burdens on healthcare providers.
Scientists suspect there’s a connection between many common neurodegenerative diseases and malfunctioning lysosomes, or processing centers in human cells that break down proteins and eliminate debris. Now a new company called Lysosomal Therapeutics is tapping into the relationship between the two to discover and develop new treatments for rare diseases and common neurodegenerative diseases, starting […]
ZURICH (Reuters) – Swiss drugmaker Novartis said on Monday a final-stage trial of its Signifor LAR therapy allowed for greater disease control in patients with the endocrine disorder acromegaly than existing therapies. Acromegaly is caused by a benign tumor in the pituitary gland that secretes excess growth hormone, and can lead to the enlargement of […]
A Dallas biotech startup has raised money to begin clinical trials for what it hopes could be a treatment for the top genetic cause of infant mortality. AveXis is developing a viral gene therapy for infants with spinal muscular atrophy. With a new round of funding led by PBM Capital, the company is hoping to […]
In yet another example of industry’s growing interest in orphan drugs, a small Texas startup secured an impressive Series A to ramp up its work on a treatment for a rare disease called CTD. Life science investors Sante Ventures and New Enterprise Associates co-led the $14 million Series A for Lumos Pharma, which is repurposing […]
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.
With help from a health IT firm, the NIH’s Undiagnosed Disease Program hopes to make the sophisticated genetic analysis methods it developed to identify underlying causes of challenging diseases available to other institutions. UDP has seen some 700 patients in the NIH’s Bethesda, Md., facility since it started back in 2008. These are patients with […]
Amid somewhat of a renaissance in drug development for orphan diseases, another gene therapy company announced funding today for a potential treatment for a rare neurodegenerative condition called Friedreich’s ataxia (FRDA). Agilis Biotherapeutics said it’s raised $8 million and will work with synthetic biology company Intrexon Corp. to develop gene therapies and genetically modified cell […]
A Cleveland biotech startup focused on a rare genetic disease provides an interesting example of the growth of alternative funding for life science companies. Abeona Therapeutics’ just-announced $750,000 seed round, which will allow the startup to complete preclinical development of gene therapies for Sanfilippo syndrome, came not from angel or venture capital investors but from […]
Raili Kerppola had a thriving career in the pharmaceutical industry when she was diagnosed with stage four adrenal cancer in 2011. While seeking treatment at the University of Michigan, she began working with an endocrinologist named Dr. Gary Hammer and her husband, a professor of biological chemistry at UM, on researching and discovering new treatments […]
The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes.