Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced the initiation of an investigator-sponsored, adequate and well-controlled clinical trial evaluating safety, tolerability and efficacy of Firdapse® (amifampridine phosphate) as a symptomatic treatment for patients with MuSK-antibody positive myasthenia gravis (MuSK-MG).

Read the full text here: http://ir.catalystpharma.com/releasedetail.cfm?ReleaseID=953737

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Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced that Patrick J. McEnany, Chief Executive Officer and Steven Miller, Ph.D., Chief Scientific Officer/Chief Operating Officer will present at the 18th Annual BIO CEO & Investor Conference being held at The Waldorf Astoria New York. The presentation is scheduled for Tuesday, February 9, at 9:30 AM ET.  The presentation materials will be posted at www.catalystpharma.com in the Investors section under Events & Presentations.

Read the full text here: http://ir.catalystpharma.com/releasedetail.cfm?ReleaseID=953336

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What is Rare Disease Day?

Rare Disease Day takes place on the last day of February each year.

The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives.

The campaign targets primarily the general public and also seeks to raise awareness amongst policy makers, public authorities, industry representatives, researchers, health professionals and anyone who has a genuine interest in rare diseases.

Since Rare Disease Day was first launched by EURORDIS and its Council of National Alliances in 2008, thousands of events have taken place throughout the world reaching hundreds of thousands of people and resulting in a great deal of media coverage.

The political momentum resulting from Rare Disease Day also serves advocacy purposes. It has notably contributed to the advancement of national plans and policies for rare diseases in a number of countries.

Even though the campaign started as a European event, it has progressively become a world phenomenon, with the USA joining in 2009, and participation in over 80 countries around the world in 2015. Hundreds of cities continue to take part in the event and we hope many more will join in 2016. Some countries have decided to raise rare disease awareness further, for example, Spain declared 2013 as the National Year for Rare Diseases.

Our objective is for the World Health Organization to recognize the last day of February as the official Rare Disease Day and to raise increasing awareness for Rare Diseases worldwide.

On rarediseaseday.org you can find information about the thousands of events happening around the world to build awareness for people living with a rare disease and their families. If you are planning an event, register your event details on our Post your Event page to get your event listed on the site.

 

 

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Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, announced today the completion of its rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Firdapse® (amifampridine phosphate) for the treatment of Lambert Eaton myasthenic syndrome (LEMS) and congenital myasthenic syndromes (CMS). Both of these diseases are ultra-rare, with prevalences in the United States believed to be approximately 3,000 and 1,000-1,500 patients, respectively.

Read the full text here: http://ir.catalystpharma.com/releasedetail.cfm?ReleaseID=947400

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Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced top line results from its Phase 1(b) double-blind, placebo controlled safety and tolerance study of CPP-115 in normal healthy volunteers. The results showed significant increases in brain levels of the surrogate marker for potential efficacy, gamma-aminobutyric acid (GABA). The main adverse effect of prolonged elevated brain GABA, somnolence, was also observed.

Read the full text here: http://ir.catalystpharma.com/releasedetail.cfm?ReleaseID=947239

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Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced that Patrick J. McEnany, Chief Executive Officer, and Steven Miller, Ph.D., Chief Operating Officer and Chief Scientific Officer, will present at the 27th Annual Piper Jaffray Healthcare Conference being held at The New York Palace, NY, December 1-2. The presentation is scheduled for Wednesday, December 2nd at 1:30 pm EST. The presentation materials will be posted at http://www.catalystpharma.com in the Investors section under Events & Presentations.

Read the full text here: http://ir.catalystpharma.com/releasedetail.cfm?ReleaseID=944064

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Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today reported financial results for the third quarter and nine months ended September 30, 2015.

“The third quarter was a very productive period for the Catalyst team, with the initiation of the rolling submission of our New Drug Application for the use of Firdapse® in the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS)”, said Patrick J. McEnany, Chairman and Chief Executive Officer of Catalyst. “We expect to complete the submission this quarter and at that time will be requesting a priority review.”

Read the full text here: http://ir.catalystpharma.com/releasedetail.cfm?ReleaseID=941720

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Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced that it will release Third Quarter 2015 financial results after market close on Monday, November 9, 2015. Further, Catalyst management will host an investment-community conference call at 8:30 a.m. EST on Tuesday, November 10, 2015 to discuss the financial results and to provide a corporate update.

Read the full text here: http://ir.catalystpharma.com/releasedetail.cfm?ReleaseID=940463

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Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced that Barbara Coffey, MD, MS, Chief, Tics and Tourette’s Clinical and Research Program, Icahn School of Medicine at Mount Sinai, will present a poster at the 62nd Annual Meeting of the American Academy of Child and Adolescent Psychiatry being held at the Henry B. Gonzalez Convention Center in San Antonio, Texas from October 26-31. The poster, entitled, “Vigabatrin in Patients with Treatment-Resistant Tourette’s Disorder: A Proof-of-Concept Study”, (Poster # NRPOSTER-2.23) will be presented at the conference from 10:00 am to 12:30 pm CDT on October 29th. The poster will be posted at www.catalystpharma.com in the Investors section under Events & Presentations.

Read the full text here: http://ir.catalystpharma.com/releasedetail.cfm?ReleaseID=938609

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Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases, today announced that Patrick J. McEnany, Chief Executive Officer, and Steven Miller, Ph.D., Chief Operating Officer and Chief Scientific Officer, will present at the 14th Annual BIO Investor Forum being held at Parc 55 San Francisco hotel, California, October 20-21. The presentation is scheduled for Wednesday, October 21st at 2:00 pm (PDT). The presentation materials will be posted at www.catalystpharma.com in the Investors section under Events & Presentations.

Read the full text here: http://ir.catalystpharma.com/releasedetail.cfm?ReleaseID=937182

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