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FDA approvals, research breakthroughs, clinical trials, and advocacy updates

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64 articles from the last 7 days

AdvocacyRSS5 days ago

Collaboration aims to improve design of FSHD clinical trials

Three organizations that work with facioscapulohumeral muscular dystrophy (FSHD) patients are joining together to make clinical trials better. FSHD is a rare muscle disease that causes weakness in the face, shoulders, and upper arms. This partnership wants to improve how these trials are designed so they can test new treatments more effectively.

WHY IT MATTERSBetter-designed clinical trials mean faster progress toward treatments for FSHD, and patient input through the FSHD Society ensures trials are structured in ways that actually work for people living with the disease.
Good to knowFacioscapulohumeral muscular dystrophyRead →
PolicyRSS5 days ago

Understanding genetic counseling in ALS

Doctors are now recommending that all ALS patients get genetic testing to understand if their disease is inherited. A genetic counselor is a specialist who helps explain what these test results mean and how they might affect family members. Understanding your genetic information can help you and your doctor make better decisions about your care and family planning.

WHY IT MATTERSIf you have ALS, genetic testing and counseling can reveal whether your condition is hereditary, which affects whether your relatives should be screened and what treatment options might work best for you.
💬 Ask your doctorAmyotrophic Lateral SclerosisRead →
AdvocacyRSS5 days ago

Patient-led group launches network of clinics to improve LGMD care

A patient-led nonprofit called The Speak Foundation has created a network of specialized clinics called LGMD Centers of Excellence to help people with limb-girdle muscular dystrophy (LGMD), a rare muscle disease. This new network aims to solve a major problem: patients with LGMD have had trouble getting consistent care and doctors have struggled to develop new treatments because the patient population is very small and spread out.

WHY IT MATTERSThis coordinated clinic network could help LGMD patients access specialized care in one place and speed up the development of new treatments by making it easier for researchers to find and study patients.
Good to knowLimb-girdle muscular dystrophy (LGMD)Read →
AdvocacyRSS5 days ago

Delphine Andrews – Life coach and disability advocate with SMA

Delphine Andrews, a life coach and disability advocate from North Carolina who lives with SMA (spinal muscular atrophy), shared her personal story in a podcast interview. She discussed how she learned to accept herself, help others with disabilities, and build a career as a life coach. The episode also covered important topics like managing caregivers and understanding how different parts of someone's identity intersect.

WHY IT MATTERSHearing from someone living with SMA about practical strategies for self-acceptance, caregiver management, and career transition can help newly diagnosed patients and families navigate similar challenges in their own lives.
Good to knowspinal muscular atrophyRead →
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