New drug for Niemann-Pick disease type C1 appears safe, effective
Researchers report that an experimental drug appears to be safe and effective at slowing the progression of Niemann-Pick disease type C1. (Source: Health News - UPI.com)
Source: Health News - UPI.com - August 11, 2017 Category: Consumer Health News Source Type: news
Experimental treatment for Niemann-Pick disease type C1 appears safe, effective
NIH-led clinical trial suggests that drug slows progression of rare neurological disease. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - August 11, 2017 Category: American Health Source Type: news
Groundbreaking drug could treat childhood Alzheimer's
Results of a small clinical trial in Washington State have shown promise to treat the neurogenerative condition called Niemann-Pick type C (NPC) that typically kills those afflicted before they turn 20. (Source: the Mail online | Health)
Source: the Mail online | Health - August 10, 2017 Category: Consumer Health News Source Type: news
CTD Holdings begins dosing in Phase I/II trial of Trappsol Cyclo to treat NPC
CTD Holdings has commenced patient dosing in a Phase I/II clinical trial of its intravenous drug candidate Trappsol Cyclo for the treatment of patients in Europe with Niemann-Pick disease type C (NPC). (Source: Drug Development Technology)
Source: Drug Development Technology - July 20, 2017 Category: Pharmaceuticals Source Type: news
NIH scientists find rare disease clues in cell ’s recycling system
The work could lead to a new generation of potential therapies for Niemann-Pick type C1 and similar disorders. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - July 17, 2017 Category: American Health Source Type: news
NIH scientists find rare disease clues in cell's recycling system
(NIH/National Center for Advancing Translational Sciences (NCATS)) Scientists have demonstrated how an investigational drug works against a rare, fatal genetic disease, Niemann-Pick type C1 (NPC1). They found that a closely related compound will activate an enzyme, AMPK, triggering a cellular 'recycling' system that helps reduce elevated cholesterol and other accumulated fats in the brains and livers of NPC1 patients, which are hallmarks associated with severe neurological problems. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 17, 2017 Category: International Medicine & Public Health Source Type: news
CTD Holdings starts enrolment in Phase I/II trial of Trappsol Cyclo for NPC
US-based biotechnology firm CTD Holdings has started patient enrolment in the Phase I/II clinical trial of Trappsol Cyclo to treat patients suffering from Niemann-Pick disease type C (NPC) in Europe. (Source: Drug Development Technology)
Source: Drug Development Technology - June 22, 2017 Category: Pharmaceuticals Source Type: news
Boy diagnosed with childhood Alzheimer's on Mother's Day
Boston-born Purnell Sabky, one, was diagnosed with Niemann-Pick disease type A on May 12. The condition is also known as childhood Alzheimer's and there is no cure. (Source: the Mail online | Health)
Source: the Mail online | Health - June 1, 2017 Category: Consumer Health News Source Type: news
Sucampo just bought this local rare disease company for $200M
Rockville global biopharmaceutical company Sucampo Pharmaceuticals Inc. has purchased Gaithersburg-based rare disease company Vtesse Inc. for $200 million, according to an announcement Monday morning.
The deal gives Sucampo access to privately held Vtesse’s treatment for the rare Niemann-Pick disease Type C1, which is still in development. The disease is a genetic mutation that causes the loss of a particular enzyme in the body, which is always fatal after early childhood.
Vtesse’s fully e nrolled… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - April 3, 2017 Category: Biotechnology Authors: Andy Medici Source Type: news
Sucampo Acquires Vtesse Inc.
ROCKVILLE, Md., and GAITHERSBURG, Md., April 03, 2017 -- (Healthcare Sales & Marketing Network) -- Sucampo Pharmaceuticals, Inc. (Sucampo) (SCMP), a global biopharmaceutical company, and Vtesse Inc. (Vtesse), a privately-held rare disease company, today an... Biopharmaceuticals, Mergers & Acquisitions Sucampo Pharmaceuticals, Vtesse, Niemann-Pick Disease, AMITIZA, RESCULA (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 3, 2017 Category: Pharmaceuticals Source Type: news
[Report] Lysosomal cholesterol activates mTORC1 via an SLC38A9 –Niemann-Pick C1 signaling complex
The mechanistic target of rapamycin complex 1 (mTORC1) protein kinase is a master growth regulator that becomes activated at the lysosome in response to nutrient cues. Here, we identify cholesterol, an essential building block for cellular growth, as a nutrient input that drives mTORC1 recruitment and activation at the lysosomal surface. The lysosomal transmembrane protein, SLC38A9, is required for mTORC1 activation by cholesterol through conserved cholesterol-responsive motifs. Moreover, SLC38A9 enables mTORC1 activation by cholesterol independently from its arginine-sensing function. Conversely, the Niemann-Pick C1 (NPC1...
Source: ScienceNOW - March 23, 2017 Category: Science Authors: Brian M. Castellano Source Type: news
Families affected by rare genetic condition that causes Alzheimer's in childhood are given new hope by what could be the first treatment
Niemann-Pick Type C is a progressive and fatal disorder that gradually robs children of the ability to speak and walk, and causes intellectual decline and memory loss. (Source: the Mail online | Health)
Source: the Mail online | Health - December 19, 2016 Category: Consumer Health News Source Type: news
Sanofi Genzyme begins enrolment in Phase II/III ASCEND clinical trial of olipudase alfa in UK
Sanofi Genzyme has begun enrolment for its pivotal Phase II/III ASCEND clinical trial of olipudase alfa to treat non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B (NPD B). (Source: Drug Development Technology)
Source: Drug Development Technology - November 7, 2016 Category: Pharmaceuticals Source Type: news
Potential treatment for Niemann-Pick type C disease
Researchers have improved a potential treatment for a rare genetic disease, decreasing its negative toxic effects by threading it onto a dumb-bell-shaped chain and holding it in place until it reaches its target. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - November 2, 2016 Category: Science Source Type: news
[In Depth] Battle over rare disease drug ensnares NIH
A bitter fight involving the National Institutes of Health (NIH), two companies, and opposing camps of researchers and parents is marring the emergence of a promising therapy for children with a rare, inherited, and uniformly fatal disorder of cholesterol metabolism. The 5-year-old National Center for Advancing Translational Sciences (NCATS), part of NIH, shepherded the early development of a cyclodextrin—a doughnut-shaped sugar molecule—as a treatment for Niemann-Pick type C-1 (NPC). Not long ago, NCATS handed the drug off to a small Maryland company, Vtesse, for further human trials and, hopefully, commercialization...
Source: ScienceNOW - October 6, 2016 Category: Science Authors: Meredith Wadman Tags: Biomedicine Source Type: news
