On Thursday, September 24, the RARE Foundation and Expecting Health co-hosted the 8th Annual Newborn Screening Bootcamp!

Original title: “RECAP: 8th Annual Newborn Screening Bootcamp ”

Every missed patient in a rare disease clinical trial represents more than a recruitment challenge, it represents a missed opportunity to advance research for communities with limited treatment options. With more than 10,000 known rare diseases and only a small percentage with approved treatments, ... Every missed patient in a rare disease clinical trial represents more than a recruitment challenge, it represents a missed opportunity to advance research for communities with limited treatment options. With more than 10,000 known rare diseases and only a small percentage with approved treatments, the need for continued research is clear. Awareness remains one of the largest barriers in rare disease research. Patients, caregivers, and even healthcare providers may not recognize symptoms, connect those symptoms to a specific condition, or know that a clinical trial is available. Patient hesitancy is another significant barrier. For many rare disease patients, joining a trial may mean reconsidering a treatment routine that took years to establish. Complex protocols and restrictive eligibility criteria can make trial participation difficult for patients to fully understand. Patient-friendly education, online pre-screening, study videos, brochures, and clear site talking points can reduce uncertainty and help potential participants understand what to expect. These tools also allow sites to focus their time on the most qualified and informed referrals. Rare disease trials frequently compete for a limited patient population, making differentiation essential.

Original title: “Overcoming the Greatest Barriers in Rare Disease Patient Recruitment | Medpace”

The Speak Foundation is a patient-led nonprofit advancing care, research, advocacy and innovation for people living with limb-girdle muscular dystrophy (LGMD) and other neuromuscular rare diseases. 1, 2026 /PRNewswire/ -- The Speak Foundation is pleased to announce a $300,000 commitment over two years from BridgeBio Pharma, Inc. to support and expand its national LGMD Centers of Excellence program, a patient-led initiative designed to strengthen specialized care, research readiness and access to emerging treatments for people living with limb-girdle muscular dystrophy. It brings patients and families together with clinicians, researchers, industry and policymakers to expand access to expert care, strengthen drug development and ensure patients help shape the decisions that affect their lives. Through initiatives including the LGMD Centers of Excellence, Speak Foundation is building patient-designed solutions that can serve as a model for the broader rare-disease community. People with LGMD often must find local specialists on their own and explain a rare condition to clinicians unfamiliar with it. At an LGMD Center of Excellence, patients can connect with clinicians experienced in neuromuscular disease who can coordinate care across specialties such as neurology, cardiology and pulmonology and provide guidance on what to monitor and avoid. Expanding the network aims to bring expert LGMD care closer to more families and reduce geographic gaps in access, while bringing together leading LGMD clinical care around shared standards of care, patient education, research participation and collaboration. "This investment allows us to continue building the clinical infrastructure the LGMD community needs," said Kat Knudson, Founder and CEO of The Speak Foundation.

Original title: “The Speak Foundation Announces $300,000 BridgeBio Commitment to Advance Patient-Led LGMD Centers of Excellence”

Amsterdam, 28 September 2026Check against delivery (The published text is based on the prepared speech. Minor differences may have occurred during delivery)Good morning,…

Original title: “Executive Director speech at EMA women’s health multi-stakeholder workshop”

Based on typical fourth quarter hiring activity, biopharma job seekers should apply for open roles sooner than later. Recruiting and career coaching experts offer advice on timing, networking, interviewing and casting a wider net than usual during Q4.

Original title: “Urgency, flexibility during Q4 job search among keys to success”

Key CDMO trends shaping strategies in a shifting global market

Original title: “Investor horizons ahead of CPHI 2026”

This page provides health care providers with clinical information about infant botulism.

Original title: “Clinical Overview of Infant Botulism”

What drives meaningful employee engagement in today’s life sciences workplace? This BioSpace report explores the factors that shape how employees feel about their organizations and what employers can do to create workplaces where talent wants to stay and thrive.

Original title: “Report: What Powers Employee Engagement”

Traveling hours away from home for a multiyear clinical trial sounds manageable in a Washington conference room. It looks very different when you're the one making that trip while your body is working against you as opposed to having access to the therapy at your own hospital. CANCER TREATMENT DELAYS SURGE ACROSS US AS DOCTOR WARNS SYSTEM ‘NEEDS FIXING IMMEDIATELY’ ... That's why I want the next FDA commissioner to listen to patients like me. We don’t just read about the disease ... Traveling hours away from home for a multiyear clinical trial sounds manageable in a Washington conference room. It looks very different when you're the one making that trip while your body is working against you as opposed to having access to the therapy at your own hospital. CANCER TREATMENT DELAYS SURGE ACROSS US AS DOCTOR WARNS SYSTEM ‘NEEDS FIXING IMMEDIATELY’ ... That's why I want the next FDA commissioner to listen to patients like me. We don’t just read about the disease in books or look at it through a microscope — we live with it every day. A 25-year-old living with Duchenne muscular dystrophy explains why the Senate confirmation fight over FDA nominee Dr. Heidi Overton is personal. Duchenne muscular dystrophy life expectancy rarely extends past 25. A young man living with the rare disease urges the Senate to confirm a patient-centered FDA commissioner. For most Americans, an FDA commissioner is just another name in the news. For the 30 million Americans like me living with a rare disease, that person can decide how many options I have and how much time I have to use them. To be clear: I'm not asking the FDA to abandon science. I respect doctors, clinical trials and hard evidence.

Original title: “Patients with rare diseases need an FDA partner, not another gatekeeper | Fox News”

Fewer shots, no hour-long pharmacy runs: two families show what medicine built for real life means.

Original title: “Kids shouldn’t have to organize childhood around medicine”

The highest scores were achieved ... with patient organisations and services ‘beyond medication’. ... “This is not just a ranking, but confirmation that our commitment is recognised by those who, every day, represent and support people living with a rare disease,” explains Carla Rapaccioli, Head of Community Engagement & Communication at Sobi Italia. For the company, advocacy is not a separate ... The highest scores were achieved in the areas of patient-centred care, relationships with patient organisations and services ‘beyond medication’. ... “This is not just a ranking, but confirmation that our commitment is recognised by those who, every day, represent and support people living with a rare disease,” explains Carla Rapaccioli, Head of Community Engagement & Communication at Sobi Italia. For the company, advocacy is not a separate activity from the rest of the business: “It means working to ensure that the voices of people with rare diseases and their carers are heard and valued,” says Rapaccioli, “and it is an integral part of our sustainability strategy: creating value means generating a tangible and lasting impact for the communities we serve.” In the PatientView 2025/2026 ranking, based on the opinions of 502 organisations in 63 countries, Sobi has retained its top spot for the second year running Looking ahead, Rapaccioli is banking on a model of advocacy that is less top-down and more collaborative amongst the various stakeholders in the system: ‘Advocacy in the future will increasingly be the result of a collective effort, in which institutions, the scientific community, patient organisations and industry each play their part in identifying practical solutions to improve diagnosis, care management, access to innovation and equity of care’. The recognition of PatientView, he concludes, “reminds us that genuine relationships and collaboration with the rare diseases community are the starting point for building them”. The operational framework is the global “Unite4Rare” initiative, through which the company says it translates feedback from communities into concrete initiatives: in Italia, over the last 12–18 months, this has included campaigns such as Cl3Ar on kidney health, projects to actively engage people with immune thrombocytopenia (ITP), programmes developed with FedEmo on haemophilia, and the ‘Rare Means Care’ initiative for Rare Disease Day. However, it is in the area of access, rather than that of recognition, that Rapaccioli identifies the outstanding issues for the Italian system. ‘One of the most significant issues remains early diagnosis: for many rare conditions, the diagnostic process can still be long and complex, with a significant impact on patients and carers,’ he observes.

Original title: “Rare diseases: when patients drive the pharmaceutical industry forward - Il Sole 24 ORE”