Rare Diseases Press Releases

SMi Reports (18.08.07.2016): Abeona President & CEO to review opportunities and challenges in orphan drug development at Orphan Drugs & Rare Diseases 2016.

Rallies Researchers and Clinicians to Meet 100 New Projects Goal

Funds to Support Awareness Campaign for Rare Autoimmune Diseases

Cure SMA, the leading organization in the US dedicated to the treatment and cure of spinal muscular atrophy (SMA), has issued a statement commending the US House of Representatives Energy and Commerce Committee for their release of the 21st Century Cures.

London, UK, 14th October 2014: A new report by visiongain calculates that the molecular diagnostics market will reach $6.13bn in 2014 with revenue showing high overall growth to 2024.

SMi is proud to have Dr. Günter Harms, Market Access & Public Affairs Director at Shire, giving the opening address at this year’s Orphan Drugs and Rare Diseases conference taking place in London on October 20th and 21st 2014.

SMi reports: According to Rare Disease UK, research and development in the area of rare diseases can often provide further understanding and insight into more common diseases.

Panel of industry experts chaired by SVP Clinigen Global Access Programs offers best practices for programs enabling access for patients with unmet medical needs

SMi is proud to have Dr. Günter Harms, Market Access & Public Affairs Director at Shire, giving the opening address at this year’s Orphan Drugs and Rare Diseases conference taking place in London on October 20th and 21st 2014.

SMi Reports: Britain was looking to accelerate access to drugs for patients with rare diseases when it launched the Early Access to Medicines Initiative in March this year. This initiative will be extremely beneficial for patients.

As of January 2014, the EMA lifted the restriction on providing fee reductions to large companies developing new orphan drugs and the Early Access to Medicines Scheme was introduced.

Special Edition airing February 28th, World Rare Disease Day focuses on raising awareness and offering hope and support for Rare Disease Community as part of the “Behind the Mystery: Rare and Genetic Diseases” series

Pfizer, GlaxoSmithKline, Novo Nordisk, Findacure Foundation and many others will meet in London at SMi’s 2nd Annual Orphan Drugs & Rare Diseases Conference to share their best practices.

With the widespread acclaim of her debut novel, WHAT WE LEAVE BEHIND, Rochelle B. Weinstein has won the hearts of readers worldwide.

SMi Reports (18.08.07.2016): Abeona President & CEO to review opportunities and challenges in orphan drug development at Orphan Drugs & Rare Diseases 2016.

Rallies Researchers and Clinicians to Meet 100 New Projects Goal

Barcelona, 3 December 2015. SOM Biotech with headquarters located at the Barcelona Science Park (PCB), is announcing its very first crowdfunding campaign for the purpose of initiating new R&D projects, related to pediatric rare diseases field.

Funds to Support Awareness Campaign for Rare Autoimmune Diseases

SMi’s 4th Annual Orphan Drugs and Rare Diseases conference will be taking place on the 19th – 20th October 2015 at the Holiday Inn Regents Park Hotel in London, UK

Cure SMA, the leading organization in the US dedicated to the treatment and cure of spinal muscular atrophy (SMA), has issued a statement commending the US House of Representatives Energy and Commerce Committee for their release of the 21st Century Cures.

London, UK, 14th October 2014: A new report by visiongain calculates that the molecular diagnostics market will reach $6.13bn in 2014 with revenue showing high overall growth to 2024.

SMi is proud to have Dr. Günter Harms, Market Access & Public Affairs Director at Shire, giving the opening address at this year’s Orphan Drugs and Rare Diseases conference taking place in London on October 20th and 21st 2014.

SMi reports: According to Rare Disease UK, research and development in the area of rare diseases can often provide further understanding and insight into more common diseases.

Panel of industry experts chaired by SVP Clinigen Global Access Programs offers best practices for programs enabling access for patients with unmet medical needs

SMi is proud to have Dr. Günter Harms, Market Access & Public Affairs Director at Shire, giving the opening address at this year’s Orphan Drugs and Rare Diseases conference taking place in London on October 20th and 21st 2014.

SMi Reports: Britain was looking to accelerate access to drugs for patients with rare diseases when it launched the Early Access to Medicines Initiative in March this year. This initiative will be extremely beneficial for patients.

As of January 2014, the EMA lifted the restriction on providing fee reductions to large companies developing new orphan drugs and the Early Access to Medicines Scheme was introduced.

Special Edition airing February 28th, World Rare Disease Day focuses on raising awareness and offering hope and support for Rare Disease Community as part of the “Behind the Mystery: Rare and Genetic Diseases” series

Pfizer, GlaxoSmithKline, Novo Nordisk, Findacure Foundation and many others will meet in London at SMi’s 2nd Annual Orphan Drugs & Rare Diseases Conference to share their best practices.

With the widespread acclaim of her debut novel, WHAT WE LEAVE BEHIND, Rochelle B. Weinstein has won the hearts of readers worldwide.

First human trial for Tay-Sachs, Canavan and other rare enzyme deficiency diseases plans to begin in early 2013 thanks to NTSAD, the nation's oldest patient advocacy organization, which leads efforts to find a cure or treatment.

ACI introduces its Guide to Orphan Drugs and Rare Diseases: Maximizing Opportunities and Overcoming Stumbling Blocks in the Designation and Development Process.

A clinically challenging and under-studied subset of diseases affecting the lymphatic system and grouped under the disease spectrum lymphangiomatosis and Gorham’s disease is the focus of a special issue of Lymphatic Research and Biology.

Citing the existence of over 6,000 rare diseases Joff Masukawa, Senior Director, Government Relations and Public Policy, Shire details his role in orphan product development and explains the importance of early engagement.

Conference Brings Together Industry Leaders to Overcome Commercialization Challenges and Enhance Patient Access to Orphan Drugs

Campaign Brings Together Support from over 90 Corporations, Non-Profit Foundations, Research Institutions, Hospitals and Advocacy Groups; New Blue Jeans Denim Ribbon Unveiled To Promote Rare Disease Awareness

The Penn State Football Uplifting Athletes fifth annual Lift For Life® event was a tremendous success.