Rare Diseases

Akcea Announces Completion of Landmark Study to Assess Disease Burden in People Living with FCS

June 13, 2018

CAMBRIDGE, Mass., June 13, 2018 (GLOBE NEWSWIRE) — Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat people with serious and rare diseases, today announced completion of a new study to assess the burden of people living with Familial Chylomicronemia Syndrome (FCS) and its impact on […]

Metafora biosystems receives €3.2 million from European Commission’s H2020 SME Instrument program

June 12, 2018

Grant will allow company to launch large-scale prospective validation study of METAglut1 test to help early diagnosis of rare De Vivo disease (Glut1 deficiency syndrome) Paris, France, June 12, 2018 – Metafora biosystems, a company developing diagnostic tests to detect abnormalities in cellular energetics, today announces that it has received a European grant of €3.2 million […]

Patients as Partners: Imagining the Future of Rare Disease Therapies

May 23, 2018

This is the first installment of our new webinar series, Healthy Opinions. It is now available to watch any time as an OnDemand resource. Healthy Opinions covers important topics in the life sciences industry. We feature top thought leaders and experts in a panel format. All panelists participate using their webcam. The focus of this […]

35th Anniversary Celebration & 2018 Rare Impact Awards

April 4, 2018

NORD’s 35th Anniversary Celebration presenting the 2018 Rare Impact Awards will be held on May 17, 2018, in Washington, D.C. at the Andrew W. Mellon Auditorium. This event will celebrate the efforts to help the 30 million Americans with rare diseases who, by the very nature of their diagnoses, may be overlooked by traditional medicine, […]

Dairy Alternatives Market Expected to Expand US$ 34 Billion Globally by 2024

March 21, 2018

Marketprognosis.com Publish a New Market Research Report On “Global Dairy Alternatives Market, Consumption Volume (by Source, Region & Application) and 20 Company Profile – Forecast to 2024” which contains global key player’s survey information and forecast to 2024. Overview of the Global Dairy Alternatives Market: The Global Dairy Alternatives Market is expected to surpass US$ […]

Confronting the Challenges of Rare Disease

March 6, 2018

The Orphan Drug Act of 1983 brought increased awareness to the need for new treatments for rare disease patients and provided incentives to pharmaceutical manufacturers, including tax credits for the costs of clinical research, seven years of patent exclusivity, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees. Of the 3,500 drug […]

Achillion Raises Awareness for C3 Glomerulopathy With Launch of WeC3G™ Initiative as Part of Rare Disease Day 2018

March 2, 2018

NEW HAVEN, Conn., Feb. 28, 2018 (GLOBE NEWSWIRE) — Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN), a biopharmaceutical company focused on advancing oral small-molecule factor D inhibitors to modulate the complement alternative pathway, today announced the launch of a new patient support initiative called WeC3G™, aimed at raising awareness and understanding of C3 glomerulopathy (“C3G”) a rare and […]

Cytokinetics Joins Global Initiative to Recognize International Rare Disease Day

March 2, 2018

SOUTH SAN FRANCISCO, Calif., Feb. 28, 2018 (GLOBE NEWSWIRE) — Cytokinetics, Incorporated (Nasdaq:CYTK) today announced that it is joining the global initiative to raise awareness of Rare Disease Day®, an international campaign led by the European Organisation for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD), dedicated to elevating the public understanding […]

Audentes Therapeutics Joins Global Organizations In Recognizing Rare Disease Day® 2018

March 2, 2018

SAN FRANCISCO, Feb. 28, 2018 /PRNewswire/ — Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, today announced its support for Rare Disease Day®. The global theme of this year’s Rare Disease Day is research. Rare disease research contributes […]

Catalyst Pharmaceuticals Announces Its Continuing Support for Rare Disease Day 2018

March 2, 2018

CORAL GABLES, Fla., Feb. 28, 2018 (GLOBE NEWSWIRE) — Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced the company’s support of the 11th annual Rare Disease Day on February 28, 2018. Rare Disease Day emphasizes the importance […]

NORD to Collaborate with FDA on Pilot Patient Engagement Activity

March 2, 2018

Washington, D.C., February 28, 2018—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, has announced a collaboration with the Food and Drug Administration (FDA) on a pilot project that would help FDA medical reviewers better understand patient experiences with their rare diseases.   “We […]

Collagen Market 2018 Analysis by Application (Wound Care, Orthopedic, Cardiovascular), Source (Bovine, Porcine, Poultry, Marine), Trends and Regional Analysis | Global Forecast to 2025

March 1, 2018

Precisemarketreports.com adds new report “Collagen Market Report by Company, Regions, Types and Applications, Global Status and Forecast to 2025” which contains global key player’s survey information and forecast to 2025. Overview of the Collagen Market: The report spread across 149 pages is an overview of the Collagen Market Report by Company, Regions, Types and Applications, […]

NORD’s 35th Anniversary Celebration & 2018 Rare Impact Awards

February 28, 2018

NORD’s 35th Anniversary Celebration presenting the 2018 Rare Impact Awards will be held on May 17, 2018, in Washington, D.C. at the Andrew W. Mellon Auditorium. This event will celebrate the efforts to help the 30 million Americans with rare diseases who, by the very nature of their diagnoses, may be overlooked by traditional medicine, research […]

Shire, Microsoft and EURORDIS form Global Commission to accelerate time to diagnosis for children with rare diseases

February 22, 2018

Cambridge, Ma., Redmond, Wash., and Brussels, Belgium – February 20, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), Microsoft and EURORDIS-Rare Diseases Europe today announced a strategic alliance to address the diagnostic challenge for patients living with a rare disease. The long road to diagnosis is one of the most important issues affecting the health, […]

Novartis drug Promacta receives FDA Breakthrough Therapy designation for first-line use in severe aplastic anemia (SAA)

January 4, 2018

Data supporting designation showed over half of treatment-naïve SAA patients achieved complete response with Promacta when given with standard immunosuppressive therapy, with overall response rate of 85%[1] Promacta is the only TPO receptor agonist indicated for the treatment of patients with SAA, currently in the refractory setting Regulatory filings of the first-line indication in US […]

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