Mary Chapman, features writer —

Eurordis is welcoming a recent call for the inclusion of health priorities, including an action plan on rare disorders, in the 2024-2029 European Union (EU) strategic agenda. The call was made by 27 health ministers of the Employment, Social Policy, Health, and Consumer Affairs (EPSCO) Council, which unanimously…

Eurordis has welcomed as a “significant stride forward” the latest European Parliament plenary vote for compromise amendments to bridge the gap between accelerated scientific progress and patient care. The recent vote was in favor of proposed reforms to the European Union’s general pharmaceutical legislation, which includes proposed…

The U.S. Food and Drug Administration (FDA), in collaboration with Duke University’s Duke-Margolis Center for Health Policy, will hold a virtual public meeting on Thursday, Dec. 14 with an aim toward ultimately improving patient community engagement in the development of rare disease treatments — including ones for  aromatic l-amino…

The National Organization for Rare Disorders (NORD) is offering a new education series to help patients understand how treatments are developed and encourage them to take part in the process, the goal being to create better therapies for rare diseases like aromatic l-amino acid decarboxylase (AADC) deficiency. Its…

Global rare disease organizations emphasized the importance of universal health coverage (UHC) for people with rare diseases during a side event to a meeting of United Nations (UN) member states to accelerate progress toward the goal of univeral coverage by 2030. The event, titled “A Blueprint for Leaving…

Delaware has become the 27th state in the U.S. to establish a Rare Disease Advisory Council (RDAC) to empower and equip rare disease community members to engage state leaders in issues important to patients and their families. This latest RDAC — established by passage of Senate Bill 55 (SB…

Phreesia has acquired MediFind, which uses advanced analytics to help patients, particularly those with serious, chronic, and rare disorders such as aromatic l-amino acid decarboxylase (AADC) deficiency, find better care. Phreesia offers digital solutions to healthcare providers, life sciences companies, and other organizations  to help patients take…

For the first time, the rare disease patient advocacy organization Global Genes will hold its RARE Health Equity Forum, RARE Advocacy Summit, and other events during a single week, from Sept. 18-21 in San Diego. The global event, called Week in RARE, will also include the…

The Sanford CoRDS 13th Annual Great Plains Rare Disease Summit will focus on rare neurodevelopmental disorders such as aromatic l-amino acid decarboxylase (AADC) deficiency. The event, hosted by Sanford Research, will be held May 11-12 virtually and in person in Sioux Falls, South Dakota, and feature presentations by…

At a May 4 awards ceremony that will also mark the anniversary of its 1983 founding, the National Organization for Rare Disorders (NORD) will honor a group of rare disease innovators, advocates, and partners. This year’s Rare Impact Awards, presented at the Smithsonian National Portrait Gallery, in Washington,…