Life hadn’t been easy generally. From my childhood I always was alone, no family growing up, no guidance or support, abuse and neglect at every… Continue reading Starting over I contacted an advisor back when Healthwatch was an advocacy agency. They were helpful to some point, but then Healthwatch changed hands. I became a volunteer with Healthwatch to help make change happen for others, taking part regularly to be a voice for those who needed to be heard when it comes to accessing necessary information. Following my child’s birth there were a number of issues that occurred, 11 issues in total, from being left in a corridor following surgery, being given other patients medication, staff not following protocols, security being worked on the list just grew. A number of students and other Drs were called into the room after the Dr had said across my shoulder to a nurse, “you know where we are going here.” He got a stand, some paper, and started drawing and explaining to us the spine and the nerves, explaining all I’d told him as he drew, and explanation of where the epidural had gone in 2005 and the chemical reaction in 2007 around the same area of previously damaged nerves, to the point of my symptoms at that time. He explained, whilst holding my hand, that he was sorry I’d had this happen and that I was one of 2 patients he had medically and professionally diagnosed in this country (UK) to that date, Dec 3rd 2009.

Original title: “Starting over - Rare Disease Day 2026”

Bringing patients into the drug development process early has proven to save time, money and resources.

Original title: “Why Companies Should Pursue True Patient-Centricity”

Industry leader Steve Ubl has served as PhRMA’s CEO for more than a decade, the longest tenure of any head of the trade group.

Original title: “PhMRA’s long-standing leader Steve Ubl to leave lobby group by year-end”

Ardena has appointed Paul Edwards as Chief People Officer

Original title: “Paul Edwards”

A journey from community pharmacy to medical leadership

Original title: “Leading by example”

Some bosses stretch you. Others make work more bearable. Both can earn your loyalty. Only one is building your future. Leadership coach Angela Justice explains how to tell the difference.

Original title: “When a Good Boss Is Bad for Your Career”

The EveryLife Foundation for Rare Diseases submitted comments reflecting coalition priorities to inform the development of educational materials for the Rare Disease Innovation Hub (RDIH) and the Accelerating Rare Disease […] The post EveryLife Foundation Submits Comments for the Development of Rare Disease Educational Materials appeared first on EveryLife Foundation for Rare Diseases.

Original title: “EveryLife Foundation Submits Comments for the Development of Rare Disease Educational Materials”

Abselion has appointed Dale Gordon as Chair of the Board of Directors

Original title: “Dale Gordon”

It’s the start of a beautiful friendship, as Jess takes to the page

Original title: “Talking the talk”

A rare disease advocacy coalition on Wednesday urged the ‌Trump administration to restore regulatory clarity as it considers new leadership at the U.S. Food and Drug Administration's Center for Biologics Evaluation and Research. The Rare ​Disease Advocacy, Biotechnology, and Investor Coalition sent the letter to President Donald Trump, U.S. ​Health Secretary Robert F. Kennedy Jr, Mehmet Oz, head of the U.S. Medicare agency and FDA Commissioner Marty Makary. The coalition, which includes nearly 100 rare disease patient advocacy groups, biotech executives and investors, said ​CBER has become less flexible in overseeing rare disease clinical trials. April 1 (Reuters) - A rare disease advocacy coalition on Wednesday urged the ‌Trump administration to restore regulatory clarity as it considers new leadership at the U.S. "We believe it is of the utmost importance that the FDA chooses a leader who understands the unique challenges of rare disease ​development and respects and ​values the views ⁠of patients and physicians," the coalition said in the letter. Vinay Prasad, the center's current incumbent, is set to ​leave the FDA at the end of April after a tenure marked ​by high-profile disputes over reviews for vaccines, including Moderna's (MRNA.O), opens new tab COVID shot, gene ⁠therapies such as uniQure's (UQ1.F), opens new tab therapy for Huntington's disease and other rare disease drugs.

Original title: “Rare disease advocacy group urges Trump administration to restore FDA clarity | Reuters”

As AI reshapes deeply specialized scientific work, R&D professionals must learn to navigate the shift to a skills-centered market. The key is knowing which skills to develop and how to leverage AI as scientific modalities evolve, technologies advance and regulatory complexity increases.

Original title: “3 Career Moves R&D Professionals Must Make in a Skills-Based, AI-Driven Economy”

RISE Together: Data Sharing across the Rare Disease Ecosystem   The RISE Workshop 3 convened on March 30, 2026, to discuss optimizing data sharing for therapy development and the collaboration, infrastructure, and education opportunities to promote data sharing as […] The post The EveryLife Foundation Attends RISE Workshop 3 appeared first on EveryLife Foundation for Rare Diseases.

Original title: “The EveryLife Foundation Attends RISE Workshop 3”

Every year, patient advocates, caregivers, and researchers from across the country travel to Washington, D.C. to attend Rare Disease Week on Capitol Hill, organized by the EveryLife Foundation for Rare Diseases. They bring one shared goal – to make sure... Every year, patient advocates, caregivers, and researchers from across the country travel to Washington, D.C. to attend Rare Disease Week on Capitol Hill, organized by the EveryLife Foundation for Rare Diseases. They bring one shared goal – to make sure the voices of the rare disease community are heard by policymakers. This year, I had the opportunity to join members of the LGS community and hundreds of others who understand firsthand the challenges of living with or caring for someone with a rare disease. For many of us, advocacy is deeply personal. Support Congressional Action to Ensure Today’s Patients Will Benefit from Robust Rare Disease Treatment Pipelines · For those new to advocacy, meetings with policymakers may feel intimidating at first, but what quickly becomes clear is that personal stories matter. The week began with advocacy training sessions and community-building events. These sessions are great for first-time attendees, preparing them to meet with congressional offices and discuss policies that can make a real difference in their lives. A one-day legislative conference was held on February 25th, followed by a Hill Day on the 26th. During the conference, leaders in the rare disease community, researchers, and policymakers spoke to the importance of research funding, access to care, and the urgent need for policies that support patients and caregivers. Advocacy doesn’t end in Washington. It continues in our communities every time we raise our voices for those living with rare diseases, in our schools when we advocate for inclusive special education services, and in our healthcare settings when we speak on behalf of our loved ones unable to speak for themselves.

Original title: “2026 Rare Disease Week on Capitol Hill - LGS Foundation”

She will provide strategic counsel to the NIH Director and other key leaders within NIH, in addition to managing process, operations, and information flows.

Original title: “Dr. Elisabeth Armstrong named NIH Chief of Staff ”

Information about meningococcal disease, including who's at risk and prevention strategies.

Original title: “Clinical Overview of Meningococcal Disease”

Watch this critical discussion on how regulators and biotech companies can bridge the expectation gap during the regulatory review process to ensure novel therapies reach patients in a timely manner—because patients don’t have time to wait.

Original title: “Webinar: Aligning With the FDA on a Regulatory Pathway To Avoid Decision Day Surprises”

Former ACIP vice chair Robert Malone claimed that Andrew Nixon, spokesperson for the Department of Health and Human Services, “trashed” him with the media, adding that he resigned because “I do not like drama.”

Original title: “After Speaking out of Turn, Malone Steps Down From Vaccine Committee”