Navigation Links
FDA Grants Orphan Drug Designation for 3,4-DAP for LEMS
Date:11/30/2009

to ten per million, or approximately 2,000 to 5,000 patients in the E.U. and 1,200 to 3,100 patients in the U.S. Approximately 50 percent of LEMS patients diagnosed have small cell lung cancer.

Patients with LEMS typically present with fatigue, muscle pain and stiffness. The weakness is generally more marked in the proximal muscles particularly of the legs and trunk. Other problems include reduced reflexes, drooping of the eyelids, facial weakness and problems with swallowing. Patients often report a dry mouth, impotence, constipation and feelings of light headedness on standing. On occasion these problems can be life threatening when the weakness involves respiratory muscles. A diagnosis of LEMS is generally made on the basis of clinical symptoms, electromyographic testing and the presence of autoantibodies against voltage gated calcium channels.

Current treatment of LEMS can consist of strategies directed at the underlying malignancy if one is present. Unfortunately, therapy of small cell lung cancer is limited and outcomes are generally poor. Immunosuppressive agents have been tried but success is limited by toxicity, and difficulty administering the regimens. A mainstay of therapy has been 3,4-DAP but its use in practice has been limited by the drug's availability. This problem will be addressed by the introduction of BioMarin's product.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan®
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2009 PR Newswire.
All rights reserved

Page: 1 2 3 4

Related biology technology :

1. Mirus Bio Awarded Grants & Contracts of Over $5 Million
2. FDA Grants Orphan Drug Designation to TREANDA, an Investigational Treatment for Chronic Lymphocytic Leukemia
3. Anesiva Grants Specific-Use License of Its Needle-Free Drug Delivery Technology to Particle Therapeutics for Diabetes Drug
4. NIH grants $750,000 to develop device to determine temperature of neonates brain
5. Edwards Lifesciences Grants UC Irvine $5 Million to Establish the Edwards Lifesciences Center for Advanced Cardiovascular Technology
6. Radius Grants Major Pharmaceutical Company Exclusive Option to License BA058 for Osteoporosis
7. Poniard Pharmaceuticals Announces European Commission Grants Orphan Medicinal Product Designation to Picoplatin for Treatment of Small Cell Lung Cancer
8. CV Therapeutics Reports Option Grants Under Nasdaq Marketplace Rule 4350
9. NASDAQ Panel Grants Merge Technologies Additional Time to Regain Compliance
10. FDA Grants Resverlogix Approval to Commence a Phase 1 Clinical Trial for RVX-208
11. Maryland Stem Cell Commission Issues Nationwide Request for Applications for New Post-Doctoral Fellowship Grants
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/15/2014)... January 15, 2014 This webinar ... on nonclinical and clinical safety assessment in biosimilars. ... guide a path for biosimilar drug development, however ... of biosimilarity with regards to quality, safety and ... of biosimilar drug development and registration, development strategies ...
(Date:1/15/2014)... NC (PRWEB) January 15, 2014 ... an enhancement to its Online Web Portal for Life ... now have the ability to specify the subject matter ... nearly 50 life science sub-domains. This will help reduce ... and scientific fields, and decrease the likelihood of error. ...
(Date:1/14/2014)... Doylestown, PA (PRWEB) January 14, 2014 ... – 11 p.m. , Location: Warrington Country Club, 1360 ... Hepatitis B Foundation, the only national nonprofit organization solely ... improving the quality of life for those affected worldwide, ... 11 at Warrington Country Club in Warrington, Pa. ...
(Date:1/14/2014)... York, NY (PRWEB) January 14, 2014 ... initiated coverage of Alliqua, Inc. (OTCQB: ALQA). Alliqua ... and marketing proprietary products to serve the wound ... , In late 2012, Alliqua was restructured ... launched the company’s new strategy to become a ...
Breaking Biology Technology:Xtalks Life Sciences Webinar Examines Safety Assessment of Biosimilars 2DTS Improves Efficiency for Life Science Document Translations 2Hepatitis B Foundation to Host Annual Crystal Ball Gala 2EquitiesIQ Initiates Coverage of Alliqua, Inc. 2EquitiesIQ Initiates Coverage of Alliqua, Inc. 3
... Partners, Inc , the global entity resulting from the ... of this year, has launched a full suite of ... relates to Dual Energy X-Ray Absorptiometry ( DXA ). ... have formally launched state-of-the-art imaging solutions to address the ...
... Calif., Oct. 15 Connected ... listening to the music you love, comes to Fresno, ... 2010, the weekend before Veterans Day. ... those with combat-related post- traumatic stress. (Photo: ...
... Physicists at the University of California, Riverside have taken ... successfully achieving "tunneling spin injection" into graphene. An ... "spin." This spin comes in two forms electrons are ... and allows for more data storage than is possible with ...
Cached Biology Technology:CoreLab Partners Launches New DXA Imaging Service Offerings 2Connected! at The Fresno Revue 2Connected! at The Fresno Revue 3UC Riverside physicists pave the way for graphene-based spin computer 2UC Riverside physicists pave the way for graphene-based spin computer 3
(Date:4/15/2014)... and Sciences (KNAW) has announced the names of five ... Prizes (one million US dollars prize money in total). ... merits to science. , This year marks the 50th ... Austrian-American chemist Erwin Chargaff in 1964. , KNAW ... following laureates: , Christopher M. Dobson, John ...
(Date:4/15/2014)... In recent years, it has become clear that ... previously assumed. In addition to the well-known building ... variations play an important role. Scientists now assume ... form a second layer of information that extends ... are rather like umlauts that extend the standard ...
(Date:4/15/2014)... 15, 2014 New research, partially funded by the ... shows that consumers can improve their diets simply by ... daily meals. , In a study ... Nutrition Sciences , lead author Theresa Nicklas, DrPH, of ... Nutrition Examination Survey datasets from 2005-2010 and evaluated the ...
Breaking Biology News(10 mins):Five scientists awarded prestigious Heineken Prizes 2German Research Foundation approves new priority program in the life sciences 2Eating rice boosts diet quality, reduces body weight and improves markers for health 2Eating rice boosts diet quality, reduces body weight and improves markers for health 3
... maternal health care issues facing women in eastern Burma ... to surveys by researchers at the Johns Hopkins Bloomberg ... than 88 percent of women had a home delivery ... than 5 time as likely to receive no antenatal ...
... (Santa Barbara, Calif.) Women should go for the ... celebrations this season. While it has been known ... broccoli, cauliflower, and cabbage, can help prevent breast cancer, ... vegetables inhibit cell proliferation was unknown until now. ...
... possible applications for double-walled carbon nanotubes have excited ... renewable energy technologies. These tiny tubes, just two ... transparent, yet can still conduct electricity. This combination ... and a host of other applications. Up ...
Cached Biology News:Maternal health problems in Myanmar widespread 2UCSB scientists show how certain vegetables combat cancer 2
... Combining linear motor and ballscrew ... pre-engineered Cartesian solutions that include ... management, and motors., , ... Pass through high-flex cabling for ...
UGT1A6 (D-20)...
... T Squareds patented high efficiency dynamic valve ... With the most compact, lightweight package in ... use in portable air and gas applications., ... high efficiency. , Includes DC Motor. ...
... designed for moving loads quickly and accurately. ... output while limiting overall size. The P5S-P ... ball bearings guided on two rails., ... provide energy absorption and controlled deceleration , ...
Biology Products: