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Baked goods made from hydrolyzed wheat flour are not toxic to celiac disease patients

Baked goods made from hydrolyzed wheat flour are not toxic to celiac disease patients, according to a new study in Clinical Gastroenterology and Hepatology, the official journal of the American Gastroenterological Association Institute.

20 Jan 2011

GSK and Prosensa commence GSK2402968 Phase III study in DMD

GlaxoSmithKline and Prosensa today announced that the first patient has commenced treatment in the Phase III clinical study investigating GSK2402968, in ambulant boys with Duchenne Muscular Dystrophy (DMD), who have a dystrophin gene mutation amenable to an exon 51 skip (up to 13% of boys with DMD). Commencement of this study confirms previously announced plans to progress this asset into Phase III.

20 Jan 2011

Genethon deploys Atempo Digital Archive to optimize biotherapy research data storage resources

Atempo, a leading provider of cross-platform data protection and archiving solutions, announced today that Genethon, a French non-profit biotherapy research center, has deployed Atempo Digital Archive, Atempo's advanced file archiving platform, to optimize its storage resources.

12 Jan 2011

Eric Lancaster receives NORD grant to study stiff-person syndrome

The National Organization for Rare Disorders has awarded a research grant for the study of stiff-person syndrome, a rare acquired neurological disorder, to Eric Lancaster, MD, PhD of the University of Pennsylvania.

11 Jan 2011

Athersys completes patient enrollment in MultiStem Phase I study for leukemia

Athersys, Inc. today announced that it has completed patient enrollment of the single dose arm of a clinical study involving administration of MultiStem to patients being treated for leukemia or other blood born cancers.

7 Jan 2011

Synageva BioPharma, to-BBB to evaluate brain drug delivery technology

Synageva BioPharma Corp. and to-BBB technologies BV, the Dutch drug brain delivery company, have entered into a research collaboration to evaluate the potential of transporting therapeutic proteins across the blood-brain barrier into the central nervous system.

6 Jan 2011

Alkeus and Columbia University enter license agreement for potential therapies for dry-AMD, Stargardt disease

Alkeus Pharmaceuticals and Columbia University announced today that they have entered into a license agreement for a set of potential therapies for the treatment of dry age-related macular degeneration (dry-AMD), Stargardt disease, and other degenerative diseases of the eye. Left untreated, these conditions often lead to impaired vision and even blindness.

5 Jan 2011

Dutch scientists provide new insights into intestinal cancer through plant study

Dutch scientists have gained important new insights into intestinal cancer from studying a plant. The disease is called Peutz-Jeghers Syndrome, a hereditary disorder where people develop intestinal polyps that turn into malignant tumors.

5 Jan 2011

LFB receives positive review feedback for closing of Willfact 1000 IU Mutual Recognition Procedure

LFB received from the German regulatory authority (Paul-Ehrlich-Institute) a positive review feedback for the closing of the Mutual Recognition Procedure (MRP) of Willfact® 1000 IU with Germany as the reference member state.

22 Dec 2010

Genetic sequencing may serve as new diagnostic tool for unknown disease

A collaborative team of scientists and physicians at the Medical College of Wisconsin and Children's Hospital of Wisconsin uses genetic sequencing to identify and treat an unknown disease.

21 Dec 2010

MDA commits to invest funds in FSMA Quinazoline Program

The MDA has committed to invest funds in the FSMA Quinazoline Program at Repligen Corporation to assist in advancing the program to an IND filing with the FDA.

16 Dec 2010

Grey's Anatomy features patient with von Hippel-Lindau disease

The second episode in a three-part series on Grey's Anatomy features a patient with von Hippel-Lindau disease (VHL). The next new episode in the show is expected to air on January 6. The first episode, "Adrift and at Peace," aired Dec. 2.

14 Dec 2010

Better approaches to ovarian cancer screening could lower cancer deaths

Cancer prevention experts have long been frustrated by the lack of a meaningful way to screen women for ovarian cancer. It is a relatively rare disease that often progresses with few symptoms until it is too late for potentially curative treatments, and elevated values of the most commonly used biomarker used in screening, CA125, are also related to other disorders.

13 Dec 2010

Rule makers in health reform hot seat; Number of administration waivers from key aspect of law climbing

As rulemaking continues to be the new focus in the health law debate, media outlets report on the Obama administration use of waivers regarding a key aspect of the new law.

10 Dec 2010

NIH to create new translational science center

National Institutes of Health (NIH) Director Francis Collins on Tuesday announced the agency is moving forward on plans to create a new research center focused on translational science, after NIH's advisory board voted to create the new center, Science's "Science Insider" blog reports (Kaiser, 12/7).

9 Dec 2010

Three abstracts on Defibrotide presented at ASH Annual Meeting

Gentium S.p.A. today announced that three abstracts on Defibrotide were presented at the 52nd Annual Meeting and Exposition of the American Society of Hematology held at the Orange County Convention Center in Orlando, Florida, December 4-7, 2010.

8 Dec 2010

QLT's oral synthetic retinoid granted orphan drug designation for treatment of Leber Congenital Amaurosis

QLT Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for QLT091001, its oral synthetic retinoid, for the treatment of Leber Congenital Amaurosis (LCA). LCA is an inherited progressive retinal degenerative disease that leads to retinal dysfunction and visual impairment beginning at birth. QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of the visual retinoid cycle.

8 Dec 2010

FDA grants QLT's QLT091001 orphan drug designation for treatment of Retinitis Pigmentosa

QLT Inc. today announced that QLT091001, the Company's oral synthetic retinoid, has been granted orphan drug designation for the treatment of Retinitis Pigmentosa (RP) from the U.S. Food and Drug Administration (FDA). This follows the FDA grant of orphan drug designation for QLT091001 for the treatment of Leber Congenital Amaurosis (LCA) also on December 7, 2010.

8 Dec 2010

Expansion of newborn screening throws some families on a journey of great uncertainty

Mandatory genetic screening of newborns for rare diseases is creating unexpected upheaval for families whose infants test positive for risk factors but show no immediate signs of the diseases, a new UCLA study warns.

7 Dec 2010

First patient dosed with rFVIIIFc in A-LONG trial

Biogen Idec and Swedish Orphan Biovitrum today announced that the first patient has been dosed with the companies' long-lasting recombinant Factor VIII Fc fusion protein (rFVIIIFc) in a global registrational clinical trial. The study, called A-LONG, is an open-label, multicenter, Phase 2/3 study designed to evaluate the safety, pharmacokinetics and efficacy of rFVIIIFc in previously-treated hemophilia A patients.

6 Dec 2010

Rare Disease News and Research

Baked goods made from hydrolyzed wheat flour are not toxic to celiac disease patients

GSK and Prosensa commence GSK2402968 Phase III study in DMD

Genethon deploys Atempo Digital Archive to optimize biotherapy research data storage resources

Eric Lancaster receives NORD grant to study stiff-person syndrome

Athersys completes patient enrollment in MultiStem Phase I study for leukemia

Synageva BioPharma, to-BBB to evaluate brain drug delivery technology

Alkeus and Columbia University enter license agreement for potential therapies for dry-AMD, Stargardt disease

Dutch scientists provide new insights into intestinal cancer through plant study

LFB receives positive review feedback for closing of Willfact 1000 IU Mutual Recognition Procedure

Genetic sequencing may serve as new diagnostic tool for unknown disease

MDA commits to invest funds in FSMA Quinazoline Program

Grey's Anatomy features patient with von Hippel-Lindau disease

Better approaches to ovarian cancer screening could lower cancer deaths

Rule makers in health reform hot seat; Number of administration waivers from key aspect of law climbing

NIH to create new translational science center

Three abstracts on Defibrotide presented at ASH Annual Meeting

QLT's oral synthetic retinoid granted orphan drug designation for treatment of Leber Congenital Amaurosis

FDA grants QLT's QLT091001 orphan drug designation for treatment of Retinitis Pigmentosa

Expansion of newborn screening throws some families on a journey of great uncertainty

First patient dosed with rFVIIIFc in A-LONG trial

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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